Conferencia de resultados del 3T de 2026 de PTC Therapeutics (PTCT): Suffiance impulsa el aumento de las previsiones

Puntos clave

• PTC Therapeutics informó ingresos totales por productos y regalías en el tercer trimestre de 2026 de 358 millones de dólares, incluidos 276 millones de dólares en ingresos netos por productos y 82 millones de dólares en regalías de Evrysdi.
• Los ingresos netos por productos aumentaron más del 100% desde los 131 millones de dólares del tercer trimestre de 2025, impulsados por los 182 millones de dólares provenientes de Suffiance. Los ingresos de Suffiance crecieron más del 20% secuencialmente.
• La empresa elevó su previsión de ingresos por productos para todo el año 2026 a 960-990 millones de dólares y la previsión de ingresos totales a 1.290-1.320 millones de dólares.
• Más de 2.000 pacientes recibían Suffiance de forma activa en todo el mundo al 30 de septiembre. La dirección sigue observando una oportunidad de ventas máximas superior a 2.000 millones de dólares para el producto.
• El efectivo, equivalentes de efectivo y valores negociables alcanzaron los 2.130 millones de dólares al cierre del trimestre. La dirección prevé que PTC Therapeutics alcance el punto de equilibrio en el flujo de caja en 2026.
• PTC prevé completar la presentación de la BLA progresiva para la terapia génica contra la enfermedad de Fabry ST920 en el cuarto trimestre de 2026, con una posible aprobación y lanzamiento comercial en 2027.

Resultados financieros principales

Rendimiento comercial y operativo

El lanzamiento de Suffiance sigue siendo el principal motor de crecimiento

Suffiance generó 182 millones de dólares en ingresos globales durante el tercer trimestre, respaldado por una demanda constante en Estados Unidos y mayores contribuciones internacionales. El producto contaba con más de 2.000 pacientes activos en todo el mundo tras su primer año en el mercado.

En Estados Unidos, la demanda provino de pacientes vírgenes al tratamiento, pacientes en los que había fracasado un tratamiento anterior y un número creciente de cambios desde Kuvan comercial o genérico. La dirección señaló que la mayoría de los pacientes activos y de los ingresos de Suffiance siguen procediendo de Estados Unidos, donde más del 85% de los pacientes con PKU aún no han probado el producto.

El crecimiento internacional estuvo liderado por Japón, con inicios adicionales de pacientes en Oriente Medio, Norte de África, América Latina y Canadá. PTC está negociando el precio y el reembolso en Alemania, Francia, Italia y España. La empresa prevé que el crecimiento internacional se acelere a medida que se expanda el acceso a lo largo de 2027.

La dirección estima que hay aproximadamente 58.000 personas con PKU en sus mercados objetivo, incluidas unas 17.000 en Estados Unidos. Reiteró su confianza en una oportunidad de ventas máximas superior a 2.000 millones de dólares para Suffiance.

ST920 suma un potencial producto comercial a corto plazo

PTC adquirió la terapia génica ST920 para la enfermedad de Fabry en fase de BLA por 111 millones de dólares por adelantado, más hasta 100 millones de dólares en pagos por hitos vinculados a la aprobación regulatoria en Estados Unidos.

La empresa planea completar la presentación de la BLA progresiva en el cuarto trimestre de 2026. La dirección afirmó que la aprobación y el lanzamiento podrían producirse en 2027 y que la transacción podría resultar acreditiva durante 2027. La preparación comercial se centra inicialmente en Estados Unidos, donde la dirección identificó a más de 11.000 pacientes con enfermedad de Fabry.

Marcas maduras y regalías

La franquicia DMD aportó 74 millones de dólares. Los ingresos de Translarna fueron de 49 millones de dólares, incluida una orden del gobierno ruso, mientras que Emflaza generó 25 millones de dólares a pesar de la presencia de múltiples competidores genéricos.

Evrysdi generó 82 millones de dólares en ingresos por regalías, basándose en las ventas globales de Roche del tercer trimestre de aproximadamente 607 millones de dólares.

Hitos del pipeline

La reunión de tipo C con la FDA para el programa Votigern sobre la enfermedad de Huntington está ahora programada para el primer trimestre de 2027 debido a la agenda de la FDA. Continúa la inscripción en el ensayo de fase 3 INVEST-HD, diseñado para incluir a aproximadamente 770 pacientes sintomáticos en etapa temprana.

PTC inició el estudio global PROVE-FA de vatiguidina en la ataxia de Friedreich. Se prevé que el ensayo de un solo brazo inscriba a aproximadamente 120 pacientes de entre 7 y 21 años y tiene como objetivo respaldar la re-presentación de la NDA.

La empresa también comenzó un estudio de Fase 2A de PTC844 en artritis reumatoide, continuó el estudio de Fase 1 de PTC612 y mantiene el calendario previsto para trasladar al desarrollo clínico en 2027 el candidato de splicing PTC303 dirigido a MSH3.

Orientación de la dirección

PTC elevó su previsión de ingresos netos por productos para todo el año 2026 a 960-990 millones de dólares, lo que representa un crecimiento interanual proyectado por la dirección del 64%-69%.

Ahora se espera que los ingresos totales de 2026 alcancen los 1.290-1.320 millones de dólares. La dirección atribuyó la mayor parte del incremento a la confianza en el crecimiento continuado de Suffiance.

La empresa sigue esperando alcanzar el punto de equilibrio en el flujo de caja en 2026, respaldada por el crecimiento de los ingresos y la gestión de gastos.

La dirección advirtió que la contribución de la franquicia DMD en el cuarto trimestre podría ser inferior a la de comienzos de 2026. Emflaza afronta una presión de precios adicional por parte de genéricos, y PTC no prevé nuevas grandes órdenes de compra gubernamentales de Brasil o Rusia para Translarna durante el cuarto trimestre.

Riesgos y aspectos a vigilar

• Persistencia de Suffiance: Las interrupciones acumuladas se situaron en el rango bajo del 20%, lo que la dirección describió como cercano a un estado estacionario. Prevé que la adherencia entre los pacientes que cambien desde Kuvan sea mayor.
• Competencia de genéricos: La entrada de un nuevo genérico de Emflaza ha reducido los precios del mercado y ha afectado las expectativas de la empresa para la marca.
• Variabilidad de las órdenes gubernamentales: Los ingresos trimestrales de Translarna pueden verse afectados por compras gubernamentales grandes e irregulares, incluida la orden rusa registrada en el tercer trimestre.
• Plazos de reembolso en Europa: Las negociaciones en Alemania siguen activas, mientras que la dirección prevé acuerdos en Francia, Italia y España durante el primer semestre de 2027.
• Plazos regulatorios: La reunión sobre Votigern con la FDA se trasladó al primer trimestre de 2027 debido a la agenda de la FDA. El lanzamiento de ST920 en 2027 sigue sujeto a completar la BLA y recibir la aprobación regulatoria.

Puntos destacados de la sesión de preguntas y respuestas con analistas

La dirección caracterizó el lanzamiento de Suffiance como una transición de una fase inicial de aceleración hacia una fase de crecimiento más estable y duradero. Prevé que los ingresos de Estados Unidos sigan representando la mayor parte de las ventas máximas, aunque el número de pacientes tratados podría ser finalmente superior fuera de Estados Unidos.

Los principales factores de crecimiento en Estados Unidos incluyen el retorno a la atención médica de adultos vírgenes al tratamiento, pacientes en los que fracasó un tratamiento previo, cambios desde Kuvan y niños de nuevo diagnóstico. La dirección afirmó que aproximadamente 2.000-2.500 pacientes en Estados Unidos continúan con Kuvan comercial o genérico y podrían ser candidatos al cambio.

Sobre el ST920, la dirección afirmó que PTC puede aprovechar su infraestructura comercial ya existente para enfermedades raras, lo que limita la necesidad de un gran despliegue independiente. La empresa ha solicitado una revisión prioritaria, destacando la designación RMAT del ST920.

PTC también se mantiene abierta a nuevas oportunidades de desarrollo de negocio. La dirección enfatizó la disciplina financiera, la capacidad comercial global de la empresa y su saldo de efectivo de más de 2.000 millones de dólares.

Transcripción completa de la conferencia de resultados

Transcripción completa de la conferencia de resultados

Comentarios de la dirección

Operator

Thank you. Ladies and gentlemen, thank you for standing by. Welcome to the PTC Therapeutics third quarter 2026 earnings conference call.

[Operator Instructions]

Today's conference is being recorded. I would.

Unknown Speaker

[to] Relations, please go ahead. Good afternoon, and thank you for joining us to discuss PTC Therapeutics' third quarter 2026 corporate update and financial results. I am joined today by our Chief Executive Officer, Dr. Matthew Klein, our Chief Business Officer, Eric Pauwels, and our Chief Financial Officer, Pierre Gravier. Today's call will include forward-looking statements based on our current expectations. These statements are subject to certain risks and uncertainties, and actual results may differ materially. Please review the slide posted on our Investor Relations website in conjunction with the call, which contains information about our forward-looking statements, our most recent quarterly report on Form 10-Q, an annual report on Form 10-K filed with the SEC, as well as our other SEC filings for a detailed description of applicable risks and uncertainties that could cause our actual performance and results to differ materially from those expressed or implied in statements. Additionally, we will disclose certain non-GAAP information during this call. Information regarding our use of GAAP to non-GAAP financial measures and reconciliation of GAAP to non-GAAP are available in today's earnings release. I will now pass the call over to our CEO, Dr. Matthew Klein.

Matthew Klein

Thank you all for joining us today. I am pleased to share the results of another quarter of record product revenue and solid execution across the company. In the third quarter, we achieved total revenue of $358 million, including $276 million in product revenue, led by continued quarter-over-quarter growth of Suffiance and sales in the U.S. and internationally. Based on this performance, we are raising our full year 2026 product revenue guidance to $960 million to $990 million and now expect total revenue of $1.29 billion to $1.32 billion. This updated guidance represents 64% to 69% year-over-year product revenue growth. In addition, with our continued effective expense management, we expect to achieve our goal of reaching cash flow break-even in 2026.

Beginning with Suffiance, we are now 1 year into the global launch, which by all measures has been a great success, and all signs point to Suffiance being a blockbuster rare disease product. In the third quarter, worldwide Suffiance revenue was $182 million, led by consistent growth in the U.S. and continued expansion internationally. As of September 30, we have over 2,000 active patients on Suffiance worldwide. In the U.S., we continue to see strong underlying demand across all patient segments. Over the course of the third quarter, we saw an uptick in branded and generic Kuvan switches with continued penetration into the therapy-naive and tried-and-failed patient segments. Adherence remains strong, reinforcing the meaningful benefits patients are experiencing on therapy, including diet liberalization, cognitive function, and mood improvements.

Internationally, we also had continued growth in the quarter, led by the strong launch in Japan, as well as new patients granted access through named patient programs across several regions. As we have discussed, we expect international revenue to continue to grow with acceleration points as we achieve access and reimbursement in additional countries over the course of 2027. In the first year of launch, we built a broad commercial patient base upon which we are well-positioned to expand as we move forward into 2027 and beyond. In the U.S., there remains a large number of patients still on branded or generic Kuvan who are candidates for switches, as well as a large number of patients without any therapy that could benefit from sapropterin. Overall, the vast majority of the 17,000 patients in the U.S. have yet to try Suffiance. We see strong underlying demand across all patient segments, fueled by the differentiated profile of Suffiance and the growing real-world evidence of meaningful Suffiance benefits.

While we are proud of all we have accomplished over the past year, we believe there is still a long way to go in the launch. Furthermore, our teams have implemented plans to address a number of the key launch learnings that will support continued success in the coming years. Internationally, we expect continued growth in 2027 as we expand into new markets in Europe, Latin America, the Middle East, Russia, and the Commonwealth of Independent States, all geographies where we have experienced teams with a track record of success. Overall, we remain confident in the $2 billion plus peak sales opportunity for Suffiance.

In the third quarter, we acquired the BLA-stage Fabry disease gene therapy asset, ST920. ST920 is a 1-time administered AAV gene therapy that provides a humanized copy of the GLA gene, enabling the body to produce sustained physiological levels of the alpha-GalA enzyme deficient in the disease. The data collected to date demonstrate that ST920 significantly increases alpha-GalA activity, decreases the lyso-GB3 substrate that causes organ damage, and improves renal function and other aspects of disease. In addition, the safety profile has been demonstrated to be favorable with no serious adverse events related to powder toxicity or other immune reactions. We expect to complete the rolling BLA submission for accelerated approval in 4Q 2026 with the potential for approval and commercial launch in 2027. Given the size of the Fabry population, our existing commercial infrastructure and experience working in the Fabry community, as well as a differentiated profile of ST920, we believe this is a potential meaningful global commercial opportunity.

Turning to the Votigern Huntington's Disease Program, due to FDA scheduling, the Type C meeting to discuss the Votigern data package and a potential path to accelerated approval is now planned for the first quarter of 2027. Enrollment continues in the Phase 3 INVEST-HD trial of Votigern, which is planned to enroll approximately 770 early symptomatic patients. The primary endpoint of the study is change in CUHDRS from baseline up to 36 months with an interim analysis planned for efficacy and futility. Excellent progress on enrollment has been made with sites now open across the globe.

For the vatiguidine Friedreich's Ataxia program, we initiated the global PROVE-FA trial in the third quarter as expected. PROVE-FA is a single-arm study with a well-matched natural history comparator arm intended to support NDA resubmission. Study design, protocol, and statistical plan have all been reviewed by FDA ahead of trial initiation. The study is expected to enroll approximately 120 individuals between 7 and 21 years of age, with the primary endpoint being the MFARS disease rating scale.

In the third quarter, we also made good progress on our other pipeline programs. We initiated a Phase 2A study of PTC844, our next generation oral DHO-DH inhibitor. As shared at our R&D day last December, PTC844 has a differentiated potency of selectivity profile that benchmarks very well against approved DHO-DH inhibitors. The Phase 2A trial is a 12-week PK-PD study being conducted in a cohort of adults with rheumatoid arthritis with the objective of confirming the relationship between dose, DHO-DH inhibition, and favorable effects on markers of B-cell and T-cell immunity. We plan to use the results of this study to help finalize which rare inflammatory disorders we will pursue for PTC844 development. The Phase 1 study of PTC612, our oral NLRP3 inhibitor, is progressing well. We have dosed several SAD and MAD cohorts, including a cohort with inflammation and cardiovascular disease that could provide early evidence of pharmacodynamic effect on a number of biomarkers. We believe PTC612's potency, selectivity, and the potential for improved tolerability are all key points of differentiation. We plan to focus development efforts on rare pulmonary indications where NLRP3 pathology has been closely linked to disease.

Finally, we continue to advance PTC303, our splicing development candidate targeting MSH3. PTC303 is our next candidate from our oral small molecule RNA splicing platform and is an example of how we are extending the reach of the platform into new and highly innovative areas of biology. By targeting somatic expansion, PTC303 has potential applications in a number of triplet repeat expansion disorders. As we have discussed, we plan to focus on DM1 as well as Huntington's disease, where we believe PTC303 can offer a complementary approach to HTT lowering. We remain on schedule for PTC303 to enter the clinic in 2027.

In conclusion, I am proud of our team's continued strong execution as we move into the fourth quarter. I will now turn the call over to Eric for a commercial update. Eric? Thanks, Matt.

Eric Pauwels

Our commercial team delivered another quarter of record product revenue, highlighted by the continued strength of the Suffiance Global Launch. Launch momentum remains robust, supported by steady growth in the U.S. and increasing contributions from international markets. In the third quarter, Suffiance revenue was $182 million, representing over 20% growth from the second quarter of 2026. More than a year into the launch, we have built a strong foundation that reinforces our confidence in our ability to deliver sustained long-term growth for the brand. Importantly, as Matt mentioned, more than 2,000 PKU patients are actively benefiting from Suffiance therapy today, providing a large growing base for future growth in 2027 and beyond.

In the U.S., underlying demand remains strong with a steady cadence of new patients and seamless reauthorizations each quarter since the launch. As a reminder, all U.S. Centers of Excellence are now prescribing Suffiance, and our field-based teams are now focused on how to penetrate deeper into each center. The teams are leveraging claims data and their prescriber interactions to tailor a center-specific approach as we build our base of knowledge targeting key PKU patient segments and roll out key programs to healthcare providers and patients going forward. We have recognized the value of organic peer-to-peer sharing of real-world experience and have built a framework, including a new ambassador program, leveraging identified Suffiance champions to share their positive experiences across multiple channels. The program is focused on the U.S. initially and will extend the concept globally where applicable. We continue to enhance our digital presence for both branded and unbranded activities, targeting messages across channels and media types to support and re-engage the PKU community through the web, social media, programmatic targeting, and live navigating PKU events.

Internationally, we saw continued momentum from the launch in Japan and continued growth in new patient starts in the Middle East, North Africa, Latin America, and Canada. In Europe, we continue to actively negotiate pricing and reimbursement in Germany, Spain, Italy, and France, with the objective of maintaining a narrow global pricing corridor. Our highly experienced market access teams are well-positioned to accelerate the global rollout and continued expansion in 2027.

As Matt mentioned, Suffiance's first year on the market has been a success by every measure, and we are just getting started. Of the approximately 58,000 individuals with PKU in target markets worldwide, the vast majority have yet to try Suffiance. Building on our first-year learnings, we have three key priorities to expand the market, reach more patients, and accelerate growth. The first priority is to continue to unlock the substantial opportunity among therapy-naive. In 3Q, more than 1 quarter of patients actively treated with Suffiance were therapy-naive adults, returning to care after years without treatment. We now better understand their treatment goals and care journeys, enabling more targeted initiatives to reach this important patient segment. The second priority is to expand adoption among patients switching from branded or generic Kuvan. Early in the launch, prescribers prioritized untreated or poorly controlled patients, including those with more challenging PKU, before broadening use among patients already on therapy.

Amplify supports switching to Palynziq. In the extension study, every responding patient achieved further PHE reductions and increased protein intake after switching. Combined with favorable U.S. payer policies and positive international health technology assessments, these data support continued expansion in this segment. The third priority is to build the real-world evidence supporting Suffiance's potential across the full spectrum of PKU severity. Physician-led studies show encouraging results in patients with classical PKU, including those with non-BH4 responsive mutations, with PA reductions and diet liberalization comparable to those seen in patients with less severe mutations. Together with the opportunity for early treatment in newborns and children, as well as treatment naive adults and patients switching from existing therapies, this growing body of evidence reinforces the science potential to address some of the most severe forms of PKU and patients with the greatest unmet need. We are applying launch learnings and executing activities that support these three priorities to expand adoption, reach more patients across the PKU spectrum, and drive sustained growth across all PKU patient segments.

While our teams are focused on the continued success of Suffiance, we are also excited about the potential launch of our next commercial product, the ST920 Fabry Disease Asset. Many of our PTC leadership and customer-facing teams have significant experience in lysosomal storage disorders and especially Fabry disease. We are excited about the potential to deliver a 1-time administered therapy that provides the body the ability to produce continuous physiological levels of humanized alpha-gal to individuals living with Fabry disease. We are advancing our U.S. commercial preparation, including mapping claims data with Centers of Excellence. Importantly, we believe the Fabry patient community will play a key role in the treatment adoption. Our initial meetings suggest strong support for this innovative approach. In parallel, we are developing the long-term durability value proposition for payers and leveraging our established U.S. market access capabilities. As we advance towards completing our FDA submission by year-end, in parallel, we are preparing to bring ST920 to the Fabry community as quickly as possible, marking another potential significant milestone for PTC's continued growth in rare diseases.

Now, turning to our mature brands, we continue to generate meaningful revenue in the third quarter from both Emflaza and Translarna. For Emflaza, we delivered $25 million of revenue and continue to see strong brand loyalty despite multiple generics in the market. For Translarna, revenue in the quarter was driven by ongoing sales in Europe, Latin America, the Middle East and North Africa, and another government order from Russia. In summary, our customer-facing teams delivered another strong quarter, including record product revenue performance, underscoring the strength of PTC's global rare disease commercial capabilities. We remain confident in our ability to grow and establish the Vias globally as the standard of care for PKU. With that, I will now turn the call over to Pierre for a financial update. Pierre?

Pierre Gravier

Thank you, Eric. I will now share the financial highlights of our third quarter of 2026. Beginning with top line results, total products and royalty revenue for the third quarter was $358 million. Total net product revenue across the commercial portfolio was $276 million compared to $131 million for the third quarter of 2025, representing over 100% growth. Third quarter 2026 product revenue includes Suffiance net product revenue of $182 million and DMD franchise revenue of $74 million. Translarna net product revenue was $49 million, including a government purchase order from Russia. Emflaza net product revenue was $25 million. For Evrysdi, Roche achieved third quarter global revenue of approximately $607 million, resulting in royalty revenue of $82 million.

For the third quarter of 2026, non-GAAP R&D expense was $95 million, excluding $11 million in non-cash stock-based compensation expense, compared to $91 million for the third quarter of 2025, excluding $9 million in non-cash stock-based compensation expense. Non-GAAP SG&A expense was $67 million for the third quarter of 2026, excluding $13 million in non-cash stock-based compensation expense, compared to $74 million for the third quarter of 2025, excluding $10 million in non-cash stock-based compensation expense. Cash, cash equivalents and marketable securities totaled $2.13 billion as of September 30, 2026, compared to $1.95 billion as of December 31, 2025. In the third quarter of 2026, we acquired ST920 for an upfront payment of $111 million and up to $100 million in contingent milestone payments tied to U.S. regulatory approval. We view the acquisition as an opportunistic business development transaction that adds a BLA-stage asset with meaningful near-term revenue potential while leveraging our current infrastructure and supporting our broader financial objectives. This value-creative transaction also preserves significant financial flexibility to pursue additional business development opportunities and support our path to cash flow break-even and long-term sustained profitability. And I will now turn the call over to the operator for Q&A. Operator? Hello?

Operator

Thank you.

[Operator Instructions]

Preguntas y respuestas

Kristen Kluska

First question comes from Kristen Kluska with Cowen. Your line is open. I wanted to ask where you would characterize yourself in the stage of the launch of Suffiance, both in the U.S. and ex-U.S., and how we should be thinking about the growth in both regions respectively.

Matthew Klein

Hi, Kristen. Thanks for the question. So, first, let me just say how incredibly excited we are about now being at the 1-year launch mark. And I think by everyone's expectations, 1, we have delivered far more than was expected, and I think we have really redefined what the opportunity can be in PKU globally for a well-differentiated therapy. And we have done a really great job in the first year, but I think we very much believe we are still in the very early innings here. We have a very long way to go. We have talked about 2,000 patients on therapy rather, out of 58,000 globally. Take the U.S. We are thinking we probably have at least 85% of PKU patients not yet tried Suffiance, and clearly that number is much bigger outside the U.S. So in this first year, what we have really done is built a strong, broad patient base that has allowed us to really accelerate the launch, delivering strong revenue numbers in the first year, but also set the stage as we now move deeper and deeper into each of the patient segments that are now well-defined and, as Eric mentioned, have tailored programs to leverage what we see as a very significant opportunity. That is both in the U.S. and abroad, and look, we know we see a lot of people externally track prescription data and predict where this launch is going. When we look at the real data, what we see is strong underlying demand, consistent growth in the U.S., room for strong long-term durable growth in the U.S., the ability now to grow ex-U.S. as we get into additional countries, and everything we see reaffirms our confidence that we are on that trajectory for the $2 billion plus opportunity we have talked about.

Kristen Kluska

Okay, thanks. And last question for me is we have seen the FDA out with a lot of public webinars specifically focused on tailoring trials for rare disease therapies in particular. So, I am curious if you have thought about that and if you think there is any read either for programs that are currently in late-stage development as you start to think about your own pipeline and platforms as you progress that into the clinic. Thanks again.

Matthew Klein

Yes, absolutely. I think we can all agree it is a really exciting time with what is going on for rare disease at FDA, and I think this has gotten to the top of the agenda in every part of the FDA. I personally have been a part of a number of discussions, and there has been the public discussions where we are hearing the importance of CBER for rare disease, showing appropriate flexibility, that what has been the standard approach for diabetes and hypertension does not apply to rare disease, and we really need a bespoke approach for each therapy. This is something that was clearly articulated again yesterday at the rare disease framework forum. So I think, not talking about anything specific, but for us in general, I think this is really great, you know, with upcoming discussions on Votigern with the Fabry program and other important programs we have in the pipeline, being able to take part in this evolution of uniform consistency, predictability, and a true understanding of the rare disease challenges. So I think overall it is a great thing, and I am hoping that it is a high tide that raises all rare disease, you know, challenges.

Operator

votes if you will. Thank you. One moment for our next question. Our next question comes from Tazeen Ahmad with Bank of America. Your line is open.

Tazeen Ahmad

On Suffiance, as it relates to the breakdown between U.S. and ex-U.S., you know ex-U.S. you do have far more patients in total than you do in the U.S., but the price point is much lower. So, I think it would be helpful to get a sense from you, Matt, about how you are thinking about ultimately the split of contribution of sales coming from U.S. versus ex-U.S. And can you give us a little bit of color on, it has only been, you know, 4 quarters or so, but, you know, is the U.S. launch slowing? I think that is what people want to know. Thanks.

Matthew Klein

Yes, absolutely. Thanks, Tazeen. I think what we have currently is we said we have over 2,000 active patients now on therapy. The majority of those remain U.S. patients. Revenue, the majority of which still comes from the U.S. I think that is very clear in the numbers. And we believe as we continue to go into launch that probably in the end the majority, when we get to peak, the majority will come from the U.S. but with strong contributions to ex-U.S. And part of that is we talked about, you mentioned the differential price points, 17,000 patients in the U.S. And as I just mentioned, so probably over 85% of those have not tried Suffiance yet. And what we are seeing is very much what you had alluded to, I think in your recent note, in your recent survey about what the potential for durable sales momentum in the U.S. given what a lot of the KOLs you spoke with see as the potential peak penetration in their centers move on. And so that is what we are seeing. So I think while we unquestionably had an acceleration phase in the first few months of the launch, we really see ourselves now in a steady, durable growth phase. And we see that quarter over quarter. Actually, despite seasonality and some trends and the headwinds in the summer, we still delivered steady growth and we see room for continued growth and increasing growth in some quarters, but overall steady growth in the U.S. and outside the U.S., as we talked about, there will be periods of acceleration as we get into pricing and reimbursement and gain access in new markets. The story of 2Q and 3Q was driven by the strong Japan launch. And then as we move into 2027, bring more countries on board into further, we see a lot more room for growth outside the U.S. So, again, as we said, we still think we are very, very early in the U.S. We got through an acceleration phase that gave us a very strong base, I think far larger than anyone would have imagined at this stage in the U.S. launch and we have strong adherence rates and that is really the key foundation from which we are going to continue to grow this quarter over quarter, year over year and get to those numbers that we talked about in terms of peak opportunity.

Tazeen Ahmad

Okay, thanks, Matt. And maybe just to get a little bit more granular on it, when you say the majority when you are at peak will be U.S., is the magnitude going to be closer to like, I do not know, 55, 45, or is it closer to 70, 30? Okay.

Matthew Klein

I think, so just to be careful, the majority of the revenue will be U.S. The number of patients we expect will be larger outside the U.S. just given because you are talking 17,000 versus 41,000 plus minus in areas we think we can get access and pricing and investment. It is hard to have an exact number right now, Tazeen. You know, we believe it is going to be the majority. A little bit is going to determine what we get outside the U.S. in terms of access and what comes through pricing and negotiation. So it is the harder part for us to figure out is exactly what that amount is going to be outside the U.S. But again, nonetheless, we remain confident in what we talked about, the $2 billion plus opportunity. We see ourselves on that trajectory to get there by our internal metrics. And, you know, again, we have a long way to go and look forward to continuing to deliver for patients. Okay, thanks, Matt.

Operator

Thank you. One moment for our next question. Our next question comes from Benjamin Burnett with Wells Fargo. Your line is open.

Benjamin Burnett

Just want to know if you can maybe talk about what the new top line guidance contemplates. I assume you are getting some additional conviction around the science, but how much what is contemplated with regards to like the DMD franchise and some of the other elements of the product revenue base. Yes.

Matthew Klein

Hi, Ben. Thanks for the questions. So once again, we felt, based on the performance to date and what we see going forward, comfortable raising the guidance. Clearly a lot of that comes from our confidence in Suffiance and the continued growth of Suffiance as we move into the fourth quarter. The DMD franchises, as we talked about today, throughout this year has really been a story of it outperforming our expectations and I think a lot of that credit goes to our teams around the world who have done an incredible job continuing to deliver for two very, uh, let us just say, two products with a lot of headwinds. As we move into the fourth quarter, what we are seeing first on the Emflaza side, we are now seeing a new generic entry to the market that has brought down the price quite a bit. So we may very well now be getting closer to that, I will say race to the bottom, but we are starting to see the impact of generics in the marketplace and generic price. So that is obviously impacted our expectations from Emflaza. And on the Translarna side, again, in the third quarter, we had a Russian order, a Russian government order, which was a bit unexpected, but we do not expect in 4Q to have any additional large government purchase orders from Brazil or Russia. So that is a lot to say that I think we are increasing guidance, majority of that driven by our enthusiasm for Suffiance. Obviously, we will have contributions from the other products, but we do not expect the DMD franchise to contribute as it has throughout the earlier parts of '26.

Benjamin Burnett

Okay, that is super helpful. And then I also wanted to ask, so I thought that the Fabry deal was super interesting. What is the appetite going forward for additional BD?

Matthew Klein

We have talked a lot about the fact that we are aware that we have over $2 billion in the bank and the capacity from a commercial standpoint to add products globally. And we see that as a priority for top-line growth. Pierre, do you want to talk a little bit about how we are looking at BD opportunities in general?

Pierre Gravier

Yes, absolutely. I mean, look, we have a global infrastructure with capacity and we demonstrated that we know how to commercialize rare disease drugs globally, and I think that is a key strength of ours, and very unique and very rare. And we will apply the same philosophy we mentioned all along. We want to be very disciplined. We have the full flexibility to operate with the $2 billion cash balance, and we want to make sure that we create value for shareholders.

Operator

Okay, great. Thanks so much. Thank you. One moment for our next question. Our next question comes from Tejas with Barclays. Your line is open.

Unknown Speaker

Can you comment first just on any trends you are seeing in the U.S. and ex-U.S. around summer seasonality and if that impacted 3Q? And maybe from what you are seeing in 4Q, could there be an acceleration into the fall? And then just a quick second question. What is the latest you are seeing from current patients on persistency and dropout rates? Thanks.

Matthew Klein

Hi, Tejas. So on the first question, we did, as I mentioned previously, we did see some summer seasonality, but again, this is the benefit of a global market. So while we saw maybe one part of the summer vacations in certain regions, other times we did not. Despite the varied seasonality, we saw a strong quarter. And again, I think this is the benefit of a global launch. And I know, Eric, do you want to talk a little bit just about how we are seeing patterns over the summer as we move forward and...

Eric Pauwels

global contributions? Yes, in terms of global contribution, first of all, again, there was some seasonality, but we saw steady and sustained growth. We saw even in the quarter new patient adds. And importantly, we also had a very strong patient base. As Matt mentioned, more than 2,000 active patients with continuous refills and reauthorizations, and we are seeing sustained prescriptions. Now, of course, seasonality is only an effect of having patients potentially on holidays and on doctor's offices, but we have seen that move back into a nice, steady cadence. And seasonality also can play out in different geographies. We also saw some of our first prescriptions actually in Latin America and we are very in the winter months. And so a lot of this is really part of our whole global.

Matthew Klein

you know, learning and global understanding of the launch to date. And then on your second question regarding adherence, we are still seeing very strong adherence. We had mentioned last quarter that we believe we are reaching steady state in terms of discontinuations, and again this quarter we have been around low 20% cumulative discontinuations to date.

Operator

Thank you. One moment for our next question. Our next question comes from Brian Cheng with JPMorgan. Your line is open.

Unknown Speaker

Maybe just on pricing negotiations here in next U.S. regions. Can you give us an update on the final pricing agreement that will come soon in Germany and also other countries like France and Spain? Thank you.

Eric Pauwels

Hi, Brian. Let me let Eric give you an update on where we are at with pricing discussions. Yeah, Brian, thanks for the question. You know, we are continuing to have those kind of discussions. I wish I could tell you we have a final agreement. I think it is interesting because the summer months literally created a little bit of the holidays in the summer months. A little bit of the delays in the administration over at GBA, but there is nothing unexpected at this point. We are still in active negotiations with GBA on the pricing and reimbursement in Germany, and we expect the decision to come out actually fairly soon, fairly shortly, but we have nothing specific yet. The launch, however, in Germany just continues with great progress. And we have seen Centers of Excellence right now, and they continue to see broad access. We have seen young patients and older patients, and and and we are also seeing switches, um, which is an important and high value driver for the uh for the business. So Germany will continue to be a major contributor and um with a narrow corridor that we are establishing right now, France, Italy, Spain, and Southern Europe, I would say first of all in Southern Europe we are very pleased with the ongoing early access programs. Patients have been enrolling very quickly in those programs. There has been high unmet needs. Pricing and negotiation in these in these areas including HTA assessments they take a little bit longer. But we have engaged with pricing and reimbursement in all three countries and we anticipate um somewhere in the first half of 2027 for those to be finalized.

Unknown Speaker

And then if I can squeeze in 1 more, you know, from a quarter to quarter dynamics, how should we think about, you know, the number that we should project for 4Q and 1Q? You know, are there potential holiday season impact and also insurance reset dynamics that we should account for?

Eric Pauwels

Well, I think in the U.S., um, our case managers and PTC Cares will be way ahead of that. During the enrollment process, we are already reaching out to make sure that every single patient, uh, we understand what their insurance coverage is, will be able to start and begin to get ahead of any of the changes, uh, and importantly, prepare all the programs in the U.S. for uh, the reset, if you will, in January. Uh, for us, this is not something new. We went through that in January. There was already a big lesson that we learned from that. Uh, and uh, now that we have thousands of patients now that we are actively working with a new patients coming in, this is a relatively routine for our team for our case management teams to handle. So simple messages, we are way ahead of that. Great, thank you, guys.

Operator

Thank you. One moment for our next question. Our next question comes from Eric Joseph with Citi.

Eric Joseph

Just 1 on ST920, assuming approval next year. Can you orient us a little bit around how you expect that launch to go from the standpoint of facilitating market access and reimbursement, as well as how we should you might be thinking about pricing and I guess also what, you know, what maybe you can describe the type of patient who might be an early adopter here.

Matthew Klein

assuming approval. Thanks. Hi, Eric. We are obviously quite excited about the the potential of bringing ST920 to individuals in Fabry, not only in the U.S. but worldwide. As we talked about, we are just getting the final parts of the BLA together and and look forward to that submission in 4Q. As you can imagine, Eric and his team have already begun to start expanding the a number of your questions, so let me turn it over to Eric to give you a bit of detail on what we are doing now in terms of market preparation and access discussion.

Eric Pauwels

Yes, thanks for the question, Eric. You know, we are very excited about this opportunity because it is a near-term opportunity, and we are leveraging that commercial infrastructure on including myself and many others. We all have gene therapy experience, lysosomal storage disease, and a very significant amount of our teams actually have direct Fabry experience. This market is incredibly large in the U.S. alone, and that is where our focus is immediately. With over 11,000 patients, what we have already learned is that the market itself is um not well served. And I think there is a lot of patients that are still going untreated. We have a number of key work streams right now in the U.S. to prepare for that. I will get to the um your question about um market access but we have to first really clearly understand the patient segment, are they naive, are they newly diagnosed, or are they on enzyme replacement therapy, as we. And as we go through that, we have to leverage the information from the STAR study, which shows, you know, stability, improvement in some cases in renal and cardiac functions, durability and efficacy going years out, um, tolerability. And the study even demonstrated quality of life benefits, which I think will drive what patients need. But in the end, I think the value proposition that you are looking at will be segmented towards the right patient. If you actually look at the costs associated with enzyme replacement therapy and care and others, we see that there is potential cost savings to patients to the healthcare system, but more importantly, really enhance quality of life improvement for patients. And I will just say that we have had 2 meetings already with patients. And these are patient advocacy groups for Fabry, and the community is incredibly excited about having a gene therapy that um that we can bring to the market. And um we already have a significant engagement with that community right now to help support those efforts.

Operator

Great. Thanks for taking the question. Thank you. One moment for our next question. Our next question comes from Faisal Khurshid with Jefferies. Your line is open.

Faisal Khurshid

I just want to ask for the U.S. when you are characterizing this steady consistent growth trajectory that you expect, what are the key levers for that? Is the bigger lever therapy naive and what are the major unlocks to achieve that then have a follow-up?

Matthew Klein

as well, thank you. Hi, Faisal. So as we talked about, we are welcoming positioned to continue to go deeper into all of the key segments. I think early in the launch, as we have discussed, we had a larger number of patients come from the therapy naive, which was really a pleasant surprise, reaffirming that is really a component of what we see as the addressable market, as well as those who tried and failed previous therapies and then as we moved into 2Q and further into 3Q, we are beginning to see now more and more the switch dynamic come into play. And as Eric highlighted in his comments, we have been able to look carefully at what we have done in the first year and now understand not only on a segment level, but on sub-segment levels, center levels, what we need to do to continue to penetrate deeper and deeper into all of those segments. And when we talk about our enthusiasm and our enthusiasm conviction in the potential for significant growth over time in the U.S., it comes from the fact that we know there is still a large, large number of patients in each of these segments, including somewhere between 2,000, 2,500 patients still on Kuvan branded and generic that we know are all candidates for sapropterin. We know that as our continue to show all those who switch over to Suffiance do better in terms of lowering or and or protein diet liberalization and other benefits. But we have also made a number of key learnings about those who were in the therapy naive uh segment, and how can we access them? What is that patient journey like? And so what we are really now, it is really the fun part of the launch. We take the learnings from the first year, develop targeted plans so that we can leverage the opportunity, the significant opportunity we still have across all of those patient segments as we move forward.

Faisal Khurshid

Got it. That is helpful. And then just 1 quick one. Is there any context you can provide on the FDA scheduling meeting for Votigern? Obviously, we have had a little bit of a history of turbulence at the FDA. Just trying to understand if this is anything like that or if it is just like simply a calendar thing. Thank you.

Matthew Klein

Yes, absolutely. Look, you know, as everyone knows, there is the PDUFA guidelines that say if you request a Type C meeting, it is typically granted within 75 days of the request, clearly given that both Novartis and PTC guided to this meeting occurring in the second half of '26. You could imagine that the request of the meeting was submitted at a time that typically would have resulted in that meeting being in the second half of 2026. And FDA gets the meeting request. And, you know, sometimes on their end, Faisal, they have to coordinate a number of different people attending these meetings. They have clearly been quite busy at a number of rare disease initiatives. Along the way of saying this is simply a matter of FDA scheduling. There is nothing to read. And we remain excited about the opportunity to share the Votigern plan data, why we believe we have a compelling package, and hopefully leveraging the precedent of TUHY-DRS being basis of an intermediate clinical endpoint accelerated approval application in HD. Awesome. Thank you.

Operator

Thank you. One moment for our next question. Our next question comes from Joseph Thome with TD Cowen.

Joseph Thome

Maybe, Matt, on those patients that you mentioned that are still on Kuvan, maybe what is the largest reason as to why they are still on Kuvan and have not switched over yet? Is it just touch points? You know, is insurance a barrier for these patients at all? Or kind of what is going to be the key learning to get them to flip? And then in that patient segment that maybe has tried something else, um and it did not work for them, what proportion of those are still under care versus maybe have been lost to follow-up and you have to do a little bit of extra digging to bring them back into the clinic? Thank you.

Matthew Klein

Hi, Joe. Okay, I will take the first one first. I think there is not a 1-size-fits-all answer here. Part of the answer is time. These centers have a limited throughput. There is only so many individual patients they are going to see at any one time, and we have heard from a lot of the KOLs that a number of these individuals will come in, you know, maybe every 3 months, maybe every 6 months. And in the early days, the focus was getting those who were not on the therapy on the therapy. Okay, now we are switching to a stage where there is more of an interest in getting the switches done. And in each center and for each patient, it may be different in terms of the time. And it may just be getting into the clinic. In some cases, clearly there is additional payer discussions we need, but our teams have really been able to manage those situations really, really well. So access questions and payer questions become a when, not an if. And furthermore, I think what our teams are really working on are continuing to deliver the value proposition here. Do we have the if? The amplified data, which again really reinforces the benefits that individuals have when they switch from branded or generic Kuvan to Suffiance in terms of significant improvements. I think one of the other take-home points here too is that even those who may be, you somewhat controlled or moderately well controlled on brand of the generic Kuvan, when they switch over, they are finding a significant increase in diet liberalization, significant natural protein intake, which all agree is a super important thing for patients. So again, the value proposition there, it is a matter of time we are working through this and our teams again are developing targeted plans and making sure that we manage through that process of the switches and it will take terms of quantifying you know those who are in the trial and failed and and are not currently under active care. It is a little bit difficult, but what we are learning importantly, is they may not be, a lot of these folks may not be under active care in terms of seeing the physician or nurse practitioner. A lot of them are tied into dietitians in the centers. And we have also made a lot of important learnings about social media networks and how individuals PKU is very well connected to each other. And while they may not be coming to clinic, they talk to other members of the community. It is a well aggregated community. And we have had the opportunity to talk to and understand from a lot of individuals how these networks work. And that is why when folks post on social media, you know, starting to feel this, having, you know, having steak for the first time, pizza for the first time, feeling so much better, brain fog, all those things that appear on social media have incredible impact in terms of continuing to generate the market pull and getting folks to come back to clinic on their own because they want to enjoy the benefits that their peers, friends, and folks they have known for years with PKU are having. And so again, part of this is what we do, right? What do we do in terms, and what our teams do in terms of a launch? We went through the first stage, we take the learnings, understandings, networks, who are the influencers, who are the key parts, how do we support patients on the patient journey, all those different things that we can now bring to bear as we continue to grow throughout the next stage of the launch.

Operator

Sounds great. Thank you very much. Thank you. One moment for our next question. Our next question comes from Maxwell Skor with Morgan Stanley. Your line is open.

Maxwell Skor

Regarding Votigern, would an accelerated approval path change in the Phase 3 design or enrollment, given that patients may be less willing to accept placebo if a commercial drug is available? And also, is there any chance you receive priority review for ST?

Matthew Klein

Thank you. Hi, Max. On your first question on Votigern, that Phase 3 study when it was set up was really set up to have the potential to serve as a confirmatory study in the context of an accelerated approval. It was also a very thoughtfully designed study, large study. Large study population to serve as a pivotal trial is needed. It was also designed to have an internal analysis so that if there was not an accelerated approval based on the PIVOT-HD data, there is the opportunity to have success on the trial at an earlier time point in a smaller number of patients. So all those different factors were built in. Also, I will point out, this is a global trial, and I, you know, it is so... It is so impressive to see the Novartis team get this trial up and running and really do an incredible job, a machine of getting study sites up all around the globe. So even if you had an accelerated approval in the U.S., there is still going to be study sites all over the world that are going to be open and enrolling where there would not be an accelerated approval. So, cannibalizing potential enrollment really is not a consideration in that matter. In terms of the potential for prior review for ST920, clearly we requested, given several factors, given the fact that there really still is significant unmet need and several, we think, relative benefits to the differentiated profile ST920 versus other available therapies. And furthermore, it also has RMAT designation, so the potential for priority view comes along with that RMAT designation. Great, thank you very much. Very helpful.

Operator

Thank you. One moment for our next question. Our next question comes from Asim Rana with UBS. Your line is open.

Unknown Speaker

Just on Suffiance, can you remind us if the average dose per patient has moved up since the launch? And then on the Fabry program, how should we be thinking about OpEx to support the launch? Thank you.

Matthew Klein

Sure. On the first question, we said in the beginning we expected average weight, since it is weight-based dosing, we expected the average weight to be somewhere in the 45 to 50 kilogram ballpark. It has stayed there. It has been pretty stable, and that is how it has been. We get adults that come on, but we also actually recently just had a number of newborns come on, which is super exciting to see a newborn get screened for PKU and positive and then be able to go on to science right away and have optimal protein intake in those critical early stages of neurodevelopment. And I think that the newborn segment, not only in the U.S. but globally is one that we can now also think about starting to penetrate the 300 plus incident individuals in the U.S. each year and a large number, of course, internationally. But overall, again, that average weight stays uh stays about even. In terms of uh OpEx, I think as we talked about since we have the commercial infrastructure built, all those key components are there. We also have a number of experienced people, as Eric mentioned, in every part of the customer-facing organization. For Fabry, it would really be an incremental spend, an incremental build next year. Thank you so much. Thank you. Thank you so much. Thank you so much. Just a quick follow-up. How quickly do you expect the deal could become accretive?

Unknown Speaker

I am sorry, could you repeat that? How quickly do you expect the ST-920 deal could become accretive?

Matthew Klein

Look, I think one thing that is particularly attractive here in this opportunity is that for, you know, $111 million asset acquisition. We believe this could be accretive in 2027. Thank you.

Operator

Thank you. One moment for our next question. Our next question comes from Kevin with RBC Capital Markets. Your line is open.

Unknown Speaker

So maybe just back on the Suffiance, can you talk about maybe some leading indicators that you can be looking at? In particular in the U.S. to suggest that you would be likely to reach those additional target populations that you talked about at your expected rate? You mentioned potential social media interactions earlier. Are there any other potential leading indicators you could be monitoring that are quantifiable to some degree? Thank you.

Matthew Klein

Yes, absolutely, Kevin. So as we talked about earlier, we know there are a lot of folks trying to look for external leading indicators and, of course, being in the closed system now we are in, really the real data are the ones that we are seeing. You know, in addition to what we see in terms of start forms coming in, our significant extensive interactions not only with prescribers but also with the patient community and all the different local and national patient communities. The feedback that we continue to get in terms of uh demand and interest in trying Suffiance, uh folks that proactively work out, reach out to us, talk to our PTC CARES team, all of those are really important leading indicators to us, just as well as the overall population that we know is out there, as well as some of our own internal center-by-center data. We know at centers how many individuals there might be in each of these different segments, either under care who have been previously obviously in care, we understand what the timeline might be to get them in. These are all the kinds of things that we follow very closely. As Eric mentioned, one of the important things we are doing right now is having, you know, bespoke center level and even sometimes prescriber level data within each center that can also help us understand what is the potential prescribing base for that individual or that center, what is there, which how much is penetrated, what is the long way we see that they still have to go. And those are all the kinds of things we look at. Eric, anything you want to add to that and what gives us the confidence in what we are seeing on the ground? No, I think you covered it really well, and I think we are really highly focused on each one of these centers going deeper to understand what are the unmet needs with those therapy naive adults and the switches. And I think the ambassador program that we discussed is incredibly important because these are three very important pillars. We have champions, patients who have actually, on all spectrums of the disease, been able to share in many ways and engage with the community about what is happening with their fear reduction. And the consequence is not only a diet liberalization, but also, um, you know, cognitive improvements and daily life. And then we have obviously peer-to-peer connected advisory panels. So we are working right now with programs that are plugging into these Centers of Excellence to really identify, um, the unmet needs and how to bring patients back into those centers. And then very importantly, make sure they have all the information going on back out for the long-term support. These programs that we talked about both web, on web, and face-to-face and all of this are actively working with these centers and these are the ones that are really very important in defining as we penetrate further and further into this what I think is still a very untapped market. We have 17,000 centers in the country. 15,000 patients in the U.S. that are eligible for this, for Suffiance, and we have still quite a ways to go. Thank you.

Operator

Thank you. One moment for our next question. Our next question comes from Basma Radwan Ibrahim with Leerink Partners. Your line is open.

Basma Radwan Ibrahim

We have a question on Suffiance and the ex-U.S. markets. Do you see similar dynamics in the ex-U.S. market, meaning do you see the treatment naive segment and the prior treatment failures? They are the primary growth driver early in the launch that slowly shift to the switch segment afterwards, or is it different? And we do have a follow-up, please.

Matthew Klein

Yes, so the first markets that we have been in, in terms of having full access, have been Germany and Japan, and most recently Japan. And that pattern is very much the same. This desire early on to get those who have not been on a therapy on sapropterin again with very positive feedback, including for those who have classical PKU, more severe non-BH4 mutations. And then as we are moving further into the launch now, a shift is starting to look at getting individuals who are on what is known as bioptin in Japan, and known as Kuvan here, on to Suffiance. And I would also comment that I think there is probably in terms of proportion of population on Suffiance, or BH4, I think it is one of the largest proportions in Japan anywhere else internationally. So that gives us a lot of confidence as well that we have a long way to go over time in the Japan launch.

Basma Radwan Ibrahim

Great. Our follow-up is about Votigern. Have you disclosed whether you are planning to disclose the 36-month data from the PIVOT-HD trial of Votigern in Huntington's along with your partner Novartis, or is that not announced yet?

Matthew Klein

Yes, I think that is going to be Novartis' call. We have not had any discussions clearly all efforts right now around getting ready for the FDA discussion on the existing data and, of course, getting the INVEST-HD study up and running. Well, it is up and running, but getting it fully enrolled. And as we mentioned in the script, Novartis' team is doing a really, really great job of getting sites up and getting folks into that study. Great. Thank you very much.

Operator

Thank you. One moment for our next question. Our next question comes from Luke Herrmann with Baird. Your line is open.

Luke Herrmann

So just a follow-up on supply discontinuations and adherence dynamics. I think you said it was 90% renewal rate with last quarter's update. And today I think you said 20% cumulative discontinuations again, similar to last quarter. So is renewal rate holding up around 90%? And I guess building on that, is there any reason to think discontinuation rate can actually come down as you pick up more switching patients who have reliably been on another drug for years maybe? Thanks.

Matthew Klein

Yes, Luke, so the numbers are, as you said, we have seen very high prescription rates, and as we commented on discontinuations, we are getting close to that steady state in the low 20 percent now. And as you alluded to when we talked about what makes that number so impressive is that early in the launch we had folks coming in who are the therapy-naive, more severe patients. And to be able to have the success rate that we have had in those individuals is incredibly encouraging. And also as you highlighted, all of our data show, and it makes sense from basic scientific biochemical principles, that if you have had a response to getting BH4, Kuvan branded or generic, you should have a better response to this Kuvan. And obviously, someone who is switching from branded Kuvan, Kuvan has already demonstrated his or herself to be adherent to that therapy. So that is a long way of saying we expect the adherence rate in the switch patients to be much higher than what we have observed to date. Eric, any additional color you want to do on prescription patterns? Yes, and I think you covered it really well. And I think what is really driving this at an individual center level and individual patient level is the switching. And the switching is very, very important because we are trying to change the paradigm from just getting to H a theoretical goal or an established goal to now normalization. And the data uh with that Matt just described is that patients who respond to generic Kuvan or Kuvan will have uh X amount of response, 70% greater response, and many of them can potentially actually get to normalized levels. So part of our goal is to really tailor some of that data and show that to many of the healthcare providers and patients that the goal is not to stay on medical foods and stay on Kuvan or anything else, but the goal is actually normalization below 120.

Operator

Thank you. One moment for our next question. Our next question comes from Alex Nackenoff with Truist Securities. Your line is open.

Unknown Speaker

Just wondering if you could expand a bit more on your experience with the Kuvan switchers thus far. Just looking for some color that could give us some confidence that the trends you are seeing are sustainable. Just curious if these patients are coming from prescribers writing their first prescription for Suffiance or perhaps from prescribers who wanted to have more experience in the more difficult to treat patients, for example, before prescribing to stable Kuvan population. Thanks so much.

Matthew Klein

Yep. I think the success stories of the switch patients are many and are continuing to fuel patient demand to switch and physician and other prescribers' strong interest in switching. As Eric alluded to, this is really, you know, an enriched population, if you will. They are definitely going to be subset responders and going to respond really well if they have been on BH4 and even served a small amount. Seeing significantly greater lower phenylalanine reduction. We saw that in the head-to-head amplified study. That is really compelling data for any prescriber, right, to be able to have actual well-designed head-to-head intrasubject data showing the significant greater reductions. The significant increase in protein intake that we are seeing, I think some of our latest numbers or there is 100 percent greater increase in natural protein on average for those who switch, that translates to significant, significant benefits for individuals in quality of life, flexibility of diet, as well as just feeling better and getting the benefits of natural protein. And not only are our field teams out there talking about that is patient-to-patient communication on social media. We have got presentations at every local, national, international, global meeting that continue to reinforce this message. And so it is not a matter of a prescriber's first sapropterin prescription, their 50th sapropterin prescription, or their 100th. These are folks who very much understand the understand the biochemical basis of the fact that their patients are quite simply going to do better as the data are continuing to show. And there is a great deal of enthusiasm to do this. And as you can imagine, if you are on a once-a-day therapy, whether you are a prescriber, a patient, a parent, if you could switch to another once-a-day therapy that could have greater benefits, well, you know, that is a pretty strong motivating factor for everyone.

Operator

Thanks so much. And I am not showing any further questions this time. I would like to turn the call back over to CEO, Dr. Matthew Klein, for any further remarks.

Matthew Klein

Thank you all again for joining the call this evening. I am incredibly proud of our team's outstanding performance again this quarter with record product revenue, continued strong momentum in the Suffiance launch. And as we have alluded to, we see as being quite durable and feel we have a very, very long way to go as we continue on this trajectory to the $2 billion plan. We also look forward to advancing our other programs that are in the pipeline and look forward to giving an update on those in January. Thank you all.

Operator

Thank you, ladies and gentlemen. That concludes today's presentation. We thank you for your participation. You may now disconnect and have a wonderful day.

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