Ascendis Pharma A/S announced the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP (navepegritide). This investigational therapy is intended for the treatment of children with achondroplasia, a rare genetic condition causing skeletal dysplasia.
The MAA is supported by comprehensive data from three randomized, double-blind, placebo-controlled clinical trials, including the pivotal ApproaCH Trial, along with up to three years of open-label extension data. These trials demonstrated that TransCon CNP has been generally well tolerated, with no discontinuations related to the study drug.
TransCon CNP is designed to provide continuous inhibition of the overactive FGFR3 pathway in achondroplasia through once-weekly administration. The U.S. New Drug Application for TransCon CNP is currently under priority review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) target date of November 30, 2025.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.