Ascendis Pharma receives Orphan Drug exclusivity and launches Yuviwel (navepegritide) in the United States

Ascendis Pharma  announced that Yuviwel (navepegritide; developed as TransCon CNP) was granted orphan drug exclusivity by the FDA and that Yuviwel is now commercially available in the United States. Yuviwel  is the first and only once-weekly treatment approved by FDA to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and is the only one to provide continuous systemic exposure to CNP over the weekly dosing interval.

Multiple  Yuviwel  prescriptions have already been approved for reimbursement for children in the U.S. enrolled through the Ascendis Signature Access Program (A.S.A.P.), with revenue now recognized following approval of first patients and initiation of therapy. Ascendis has established A.S.A.P. to provide support to children in the U.S. prescribed  Yuviwel  and their caregivers throughout the treatment journey, including a dedicated Patient Access Liaison, programs designed to help navigate each step of the treatment process, and information about co-pay and other assistance programs for eligible patients.

“Our daughter has gained new independence and confidence with her growth, following treatment with YUVIWEL in a clinical trial,” said Brandi, mother of an 11-year-old child living with achondroplasia. “It is satisfying to know that this once-weekly treatment is now available to others in the achondroplasia community seeking pharmacological options to mitigate the potential impacts of this condition.”

“We’re excited to see the FDA grant orphan drug exclusivity for YUVIWEL, acknowledging it as a highly differentiated treatment option,” said Jan Mikkelsen, President and Chief Executive Officer of Ascendis Pharma. “We have now delivered and commercialized our third FDA-approved TransCon product in a row, and remain on track to fulfill our objective of being the leader in Growth Disorders.”