Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has accepted for Priority Review its New Drug Application (NDA) for TransCon CNP (navepegritide). This investigational therapy is for the treatment of children with achondroplasia.
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025, for the completion of its review. Importantly, the FDA also informed Ascendis that it is not currently planning to hold an advisory committee meeting to discuss this application, which can often streamline the review process.
TransCon CNP is designed to provide sustained exposure to C-type natriuretic peptide (CNP), continuously inhibiting the overactive FGFR3 pathway in achondroplasia. Clinical trials have shown that TransCon CNP demonstrated significantly higher annualized growth velocity, the primary endpoint, compared to placebo, along with multiple benefits beyond linear growth and a safety profile similar to placebo.
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