FDA grants accelerated approval for Ascendis Pharma's weekly Yuviwel for paediatric dwarfism

The FDA granted accelerated approval to Ascendis Pharma's once-weekly Yuviwel (navepegritide) to increase linear growth in children aged two and older with achondroplasia. The agency also issued Ascendis a rare paediatric disease priority review voucher with the approval.

The US Food and Drug Administration has granted accelerated approval to Ascendis Pharma's Yuviwel (navepegritide) for the treatment of achondroplasia in children. The approval covers patients aged two and older with open growth plates and is limited to increasing linear growth. The agency reviewed data from randomized, placebo-controlled trials and open-label extensions to reach its decision. Ascendis received a rare paediatric disease priority review voucher alongside the approval. Key facts: - The accelerated approval is for increasing linear growth in children aged two and over with achondroplasia and open growth plates. - The decision was based on three randomized, double-blind, placebo-controlled trials and up to three years of open-label extension data, including the Phase III ApproaCH study where Yuviwel showed higher annualised growth velocity at week 52 versus placebo. - Reported outcomes included improved lower-limb alignment, body proportionality, and positive changes in health-related quality of life, with safety described as similar to placebo. - Yuviwel is a once-weekly subcutaneous prodrug of C-type natriuretic peptide designed to provide continuous exposure to counteract overactive FGFR3 signalling in achondroplasia. - The FDA issued Ascendis a rare paediatric disease priority review voucher with this approval; Ascendis may use the voucher on another application or sell it, and such vouchers have recently fetched around $200 million. The PRV scheme was reauthorized in February 2026. - Ascendis expects to make Yuviwel available through prescribing physicians in the US in Q2 2026, and the company noted that continued approval may be contingent on verification of clinical benefit in confirmatory trials. Summary: The accelerated approval introduces a once-weekly CNP prodrug option for increasing linear growth in children with achondroplasia. Ascendis plans to make Yuviwel available in the US in Q2 2026, and continued approval will depend on results from confirmatory trials.