Ascendis Pharma announced that the Week 52 data from its randomized, double‑blind, placebo‑controlled ApproaCH trial of TransCon CNP (navepegritide) in children with achondroplasia have been published in JAMA Pediatrics. The 84‑patient study, enrolling children aged 2‑11, showed a statistically significant increase in annualized growth velocity versus placebo, along with measurable improvements in lower‑limb alignment, body proportionality, and health‑related quality of life. Safety and tolerability were comparable to placebo, with only low‑rate injection‑site reactions and no serious adverse events.
The trial results provide robust evidence that a once‑weekly biologic can deliver meaningful growth benefits in a rare disease population that has long lacked effective, convenient therapies. The statistically significant growth velocity gain, coupled with the safety profile, supports Ascendis’s claim that TransCon CNP can achieve catch‑up growth while minimizing the treatment burden that has historically limited patient adherence.
Regulatory agencies have taken note. The FDA has granted the drug priority review, with a PDUFA target date of November 30, 2025, and the European Medicines Agency is reviewing the data. The publication of the pivotal results in a high‑impact journal strengthens the company’s submission dossier and may accelerate the approval process, potentially bringing the therapy to market earlier than originally anticipated.
In the same week, Ascendis reported Q3 2025 financial results that illustrate the company’s commercial momentum. Total revenue rose to €213.6 million, up 270 % from €57.8 million in Q3 2024, driven by strong sales of YORVIPATH and SKYTROFA. Operating profit reached €11.0 million, a turnaround from a net loss in the prior year, reflecting the company’s focus on scaling its core products. However, the company posted an EPS of –€1.00, missing the consensus of –€0.28, largely because of significant net finance expenses that offset the operating gains. The revenue beat and operating profit highlight the company’s ability to generate cash from its rare‑disease portfolio, while the EPS miss underscores the impact of financing costs on profitability.
Ascendis’s TransCon technology platform, which enables once‑weekly dosing of biologics, is a key differentiator that could improve patient adherence and reduce healthcare costs. The achondroplasia market, estimated at 250,000 affected individuals worldwide, represents a sizable opportunity for a therapy that offers a convenient dosing schedule. In addition, the platform’s versatility positions Ascendis to expand into other therapeutic areas, reinforcing its long‑term growth strategy.
"TransCon CNP-treated children with achondroplasia exceeded the growth rate of the general population, suggesting that once‑weekly TransCon CNP provides catch‑up growth with safety and tolerability comparable to placebo," said Jan Mikkelsen, President and CEO. "With SKYTROFA® and TransCon CNP, Ascendis is uniquely positioned to become the leader in the treatment of skeletal dysplasias and growth disorders." Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer, added, "We are especially pleased to see these overall results of our pivotal trial for TransCon CNP, including results that go beyond linear growth, published in this prestigious journal."
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