The U.S. Food and Drug Administration extended the review of Ascendis Pharma’s New Drug Application for TransCon CNP (navepegritide) for children with achondroplasia. The original PDUFA target action date of November 30 2025 was moved to February 28 2026, a three‑month delay that pushes back the drug’s expected market entry. The extension follows the FDA’s request for additional information on a post‑marketing requirement; Ascendis submitted a major amendment on November 5 2025, which the agency classified as a major amendment.
The delay defers the first‑year revenue that Ascendis had projected for FY 2026 and the associated cash‑flow benefits. TransCon CNP is a key growth driver in the company’s rare‑disease portfolio, and the postponement will shift the timing of the expected revenue stream. Management noted that the post‑marketing study protocol revision is the only outstanding issue and that the company remains committed to a swift U.S. launch.
In the broader achondroplasia market, BioMarin’s Voxzogo is the only FDA‑approved therapy for pediatric patients. BridgeBio Pharma’s infigratinib is in development, and other candidates are in earlier stages. A three‑month delay for TransCon CNP could give competitors additional time to capture market share, potentially impacting Ascendis’s projected revenue and market position.
Ascendis’s Q3 2025 earnings, released shortly before the FDA announcement, showed revenue of $1.12 billion, up 12% YoY, driven by strong sales of YORVIPATH and SKYTROFA. Operating income rose to $210 million, reflecting disciplined cost management. EPS of $0.21 beat consensus of $0.17 by $0.04, a 24% beat, largely due to cost controls and the continued growth of its core products.
Analysts remained upbeat. TD Cowen, Cantor Fitzgerald, and Wolfe Research all maintained buy ratings, citing the post‑marketing requirement as a minor issue and expressing confidence in eventual approval. The market reaction was muted; the stock traded flat, indicating that investors viewed the extension as a procedural delay rather than a fundamental setback.
Management’s CEO Jan Mikkelsen said, “We have addressed all FDA requests and are working closely with the agency to finalize the post‑marketing requirements. We remain focused on bringing TransCon CNP to patients in the U.S. as soon as possible.” The company’s guidance for FY 2026 remains unchanged, underscoring confidence in the drug’s commercial prospects despite the delay.
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