Shares of Ascendis Pharma AS (NASDAQ:ASND) rose 5.8% after the U.S. Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) for TransCon CNP under priority review. The experimental therapy targets achondroplasia, a rare genetic condition that affects bone growth in children. A Prescription Drug User Fee Act (PDUFA) decision is scheduled for November 30, 2025.
TransCon CNP is a prodrug of C-type natriuretic peptide (CNP), designed for once-weekly dosing. Clinical trial data have shown that the therapy significantly improved annualized growth velocity compared to placebo. Notably, the treatment also demonstrated benefits beyond height, including enhancements in lower limb alignment, spinal canal development, and muscle strength – a first in achondroplasia clinical research.
The therapy’s safety profile was comparable to placebo, with no serious safety concerns reported. Dr. Aimee Shu, Ascendis Pharma’s EVP of Endocrine & Rare Disease Medical Sciences, emphasized the unmet need in this patient population and the company’s commitment to delivering a meaningful treatment option.
The FDA has opted not to convene an advisory committee, indicating a potentially smoother review process. Priority review status is reserved for treatments that may offer substantial improvements over existing therapies for serious conditions, signaling the agency’s recognition of TransCon CNP’s potential impact.
Patient advocacy groups also welcomed the development. Chandler Crews, founder of The Chandler Project and an advocate for those with achondroplasia, highlighted the hope that treatments like TransCon CNP bring for improving both health outcomes and quality of life.
Investors responded positively to the update, reflecting growing optimism that Ascendis Pharma could soon secure FDA approval for a first-in-class treatment that addresses both the physical and systemic challenges of achondroplasia.