EMA committee backs orphan exclusivity for Ascendis’ achondroplasia drug
The EMA’s Committee for Orphan Medicinal Products (COMP) has recommended that Ascendis Pharma’s once-weekly TransCon CNP (navepegritide) keep its orphan designation for achondroplasia in children. Once adopted by the European Commission, this would give the drug 10 years of market exclusivity in the EU.
November 11, 2026
The opinion follows the October recommendation from the EMA’s CHMP to approve TransCon CNP for children aged two and older whose growth plates have not closed. A final European Commission decision on the marketing application is expected in the fourth quarter of 2026. If approved, Ascendis plans its first EU launch under the brand name Yuviwel in Germany in the first quarter of 2027.
TransCon CNP is a prodrug of C-type natriuretic peptide designed to counteract the overactive FGFR3 signaling that causes achondroplasia, a genetic condition estimated to affect more than 250,000 people worldwide. It was approved by the US FDA in February 2026.
“We are pleased to see TransCon CNP recognized as a differentiated treatment able to make a major contribution to patient care for children with achondroplasia,” says President and CEO Jan Mikkelsen.
Source: Ascendis Pharma press release, 6 November 2026 — read the release
Published: November 11, 2026
