US FDA extends review of Ascendis Pharma's therapy for children with dwarfism

Nov 25 (Reuters) - The U.S. Food and Drug Administration on Tuesday extended its review of Ascendis Pharma's A71.F therapy for children with a rare genetic disorder that causes dwarfism, the company said.

The health regulator extended its review by 3 months to February 28, 2026.

The FDA extended the deadline after Ascendis submitted additional information on November 5 regarding a required follow-up study, prompting a major change to the application, the company said.

Ascendis had answered all remaining questions from the agency, including providing a revised plan for the post-approval study, and would continue to work closely with the FDA to finalize the requirements, CEO Jan Mikkelsen said.

The rare disorder, called achondroplasia, is caused by a genetic mutation that affects a protein in the body called fibroblast growth factor receptor 3, or FGFR3, resulting in dwarfism.

Ascendis' therapy, TransCon CNP, targets a naturally occurring peptide called C-type natriuretic peptide (CNP), which has been shown to counteract the growth-inhibiting effects of the FGFR3 mutation and stimulate growth.