The FDA granted accelerated approval to Ascendis Pharma’s TransCon CNP (now Yuviwel) for children two years and older with achondroplasia, using annualized growth velocity as the surrogate basis for clearance. The decision ends BioMarin’s effective monopoly in the indication and positions Ascendis to compete directly with Voxzogo, which remains the incumbent therapy. Ascendis must confirm clinical benefit in post‑marketing studies to maintain approval. The company expects to begin physician distribution in the second quarter; analysts project modest 2026 uptake with potential acceleration in 2027 and beyond, depending on confirmatory data and label expansions.
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