Ascendis Pharma: TransCon CNP Orphan Drug Designation Recommended for Maintenance in EU

Ascendis Pharma A/S announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending the maintenance of orphan medicinal product designation for its once-weekly TransCon CNP (navepegritide) for the treatment of achondroplasia in children. This designation, upon final adoption by the European Commission, would grant Ascendis 10 years of orphan market exclusivity. This follows a positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP) recommending marketing authorization for TransCon CNP in the EU. The final European Commission decision is anticipated in Q4 2026, with a potential EU launch in Germany in Q1 2027 under the brand name YUVIWEL®.