Ascendis Pharma Receives FDA Orphan Drug Exclusivity For YUVIWEL And Launches Commercial Availability In The United States

Ascendis Pharma A/S Ascendis Pharma A/S announced that the U.S. Food & Drug Administration U.S. Food and Drug Administration has granted orphan drug exclusivity to YUVIWEL (navepegritide; developed as TransCon CNP). The company also confirmed that the treatment is now commercially available in the United States. 

YUVIWEL is currently the only once-weekly therapy approved to support linear growth in children aged two years and older with achondroplasia who still have open growth plates. The treatment is designed to maintain steady, systemic exposure to CNP throughout the full weekly dosing period, offering a consistent therapeutic effect.

Several prescriptions for YUVIWEL have already been approved for reimbursement for children enrolled through the Ascendis Signature Access Program (A.S.A.P.), and the company has begun recognizing revenue as the first patients start therapy. A.S.A.P. was created to guide families and caregivers through the treatment journey by providing a dedicated Patient Access Liaison, step-by-step support for navigating insurance and treatment procedures, and information on available co-pay or financial assistance options for eligible families. 

The program also supports healthcare providers by offering clinical education and assistance with prior authorization, documentation, and insurance appeals. More details about the support program are available on the YUVIWEL website.

Families who participated in clinical research have already shared positive experiences. One parent, Brandi, whose 11-year-old daughter was treated with YUVIWEL during a trial, expressed that the treatment has helped her child gain independence and feel more confident as her growth improved. She also said that it is reassuring to know that other families in the achondroplasia community now have access to this once-weekly treatment option.

Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma, stated that the orphan drug exclusivity granted by the FDA recognizes YUVIWEL as a uniquely differentiated treatment. He also noted that this marks the company’s third FDA-approved TransCon product to reach the market, reinforcing their long-term goal of becoming a leader in growth disorder therapies.

YUVIWEL was first granted Orphan Drug Designation by the FDA in February 2019. This designation is reserved for treatments intended for rare diseases that affect fewer than 200,000 people in the United States. It provides several incentives to developers, including tax credits for clinical research, exemption from FDA user fees, and, after approval, a seven-year period of U.S. marketing exclusivity for the designated condition. For YUVIWEL, this exclusivity extends until February 27, 2033, unless certain exceptions apply.

Scientifically, YUVIWEL is a prodrug of C-type natriuretic peptide (CNP), created to deliver continuous levels of active CNP across the body. This mechanism is intended to counterbalance the overactive FGFR3 signaling that drives growth complications in achondroplasia. The FDA approved YUVIWEL on February 27, 2026, under its Accelerated Approval Program. Approval was based on improvements in annualized growth velocity, and continued authorization will depend on confirmatory trials that verify and further describe the long-term clinical benefits.