Ascendis Pharma A/S (NASDAQ: ASND) announced that YUVIWEL (navepegritide) received orphan drug exclusivity from the U.S. Food and Drug Administration and is now commercially available in the United States. The drug is approved to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses.

YUVIWEL is administered once weekly and represents the first FDA-approved treatment in this indication. The orphan drug exclusivity will run through February 27, 2033, according to the company's statement.

Multiple YUVIWEL prescriptions have been approved for reimbursement, with revenue recognition beginning following approval of first patients and initiation of therapy. Ascendis has established the Ascendis Signature Access Program to provide support to patients and caregivers throughout treatment.

The FDA approved YUVIWEL on February 27, 2026 under the agency's Accelerated Approval Program. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

YUVIWEL is a prodrug of C-type natriuretic peptide designed to provide continuous exposure of active CNP to counteract overactive FGFR3 signaling in achondroplasia. The drug received Orphan Drug Designation from the FDA in February 2019.

Achondroplasia is a rare genetic condition estimated to affect more than 250,000 people worldwide. The condition arises from a fibroblast growth factor receptor 3 variant that leads to skeletal dysplasia and can involve muscular, neurological, and cardiorespiratory complications.

Common side effects of YUVIWEL include injection site reactions and potential risk of low blood pressure. The drug carries warnings about monitoring for symptoms of decreased blood pressure during treatment.