The U.S. Food and Drug Administration approved Ascendis Pharma’s once‑weekly therapy YUVIWEL (navepegritide) for children 2 years and older with achondroplasia on February 27, 2026, under the Accelerated Approval Program based on an improvement in annualized growth velocity.
The approval was grounded in data from three randomized, double‑blind, placebo‑controlled trials and up to three years of open‑label extension data, including the pivotal ApproaCH trial published in JAMA Pediatrics. The trials demonstrated a clinically meaningful increase in growth velocity that met the surrogate endpoint required for accelerated approval.
YUVIWEL is the first and only once‑weekly treatment that delivers continuous systemic exposure to C‑type natriuretic peptide (CNP) over the entire weekly dosing interval. This differentiates it from the existing daily CNP analog, vosoritide, approved in 2021. The achondroplasia market is estimated to include more than 250,000 patients worldwide, and the once‑weekly dosing schedule is expected to improve adherence and patient experience.
The approval removes an 18‑month regulatory hurdle that had delayed the drug’s launch and allows Ascendis to begin commercial sales in the early part of the second quarter of 2026. The company also received a Rare Pediatric Disease Priority Review Voucher, which can accelerate the review of a future application. In 2025, Ascendis reported product revenue of approximately €684 million, a net loss of €228 million, and a gross margin of 86.89 %. The company projects operating cash flow of €500 million in 2026 and aims for €5 billion in global annual product revenue by 2030.
"The approval of once‑weekly YUVIWEL is a major step forward in the treatment of children with achondroplasia, giving physicians for the first time the option of prescribing a once‑weekly medicine backed by compelling efficacy and excellent tolerability data from three randomized, double‑blind, placebo‑controlled clinical trials," said Carlos A. Bacino, MD, Professor of Molecular and Human Genetics, Baylor College of Medicine and Texas Children’s Hospital.
"We are confident in YUVIWEL’s potential to transform the treatment of achondroplasia and are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone," said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. "We have listened to advocacy groups for people with dwarfism to ensure we address what the community actually cares about. This reflects our ongoing commitment to pursue outcomes that patient communities have told us are important to them, and gives the achondroplasia community a new way to look at the promise of pharmacological treatment options."
The approval marks a significant milestone for Ascendis, validating its TransCon platform and positioning YUVIWEL as a key driver of future growth in the rare‑disease portfolio. The once‑weekly dosing, combined with the drug’s proven efficacy, is expected to enhance market penetration and support the company’s broader strategy to expand its rare‑disease pipeline.
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