Ascendis Pharma A/S announced the submission of its New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon CNP (navepegritide). This investigational prodrug is intended for the treatment of children with achondroplasia, a rare genetic condition.
The NDA filing 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 treatment with TransCon CNP was associated with increased growth velocity, reduced health-related burden, stronger muscle function, and the straightening of abnormal leg bowing.
TransCon CNP is administered once weekly and is designed to provide continuous exposure of active C-type natriuretic peptide (CNP) to tissues throughout the body. The clinical data also highlighted a safety and tolerability profile comparable to placebo, reinforcing its potential as a best-in-class treatment. Ascendis remains on track to submit its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) during Q3 2025.
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