Ascendis Pharma Says COMP Of EMA Adopts Positive Opinion Recommending Maintenance Of Orphan Medicinal Product Designation For Once-Weekly TransCon CNP As Treatment For Achondroplasia In Children

Ascendis Pharma A/S (NASDAQ:ASND) today announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending maintenance of orphan medicinal product designation for once-weekly TransCon CNP (navepegritide) as a treatment for achondroplasia in children. The designation would provide Ascendis benefits that include 10 years of orphan market exclusivity upon final adoption of the COMP opinion by the European Commission (EC).

The opinion follows the EMA’s Committee for Medicinal Products for Human Use (CHMP) positive opinion recommending marketing authorization for once-weekly TransCon CNP in the European Union for the treatment of achondroplasia in children two years of age and older whose epiphyses are not closed. The final EC decision on Ascendis Pharma’s Marketing Authorisation Application (MAA) is expected during Q4 2026.

If authorized by the EC, Ascendis expects its first EU launch of TransCon CNP under the brand name YUVIWEL® to be in Germany during Q1 2027.