FDA Grants Accelerated Approval To Ascendis Pharma's YUVIWEL For Achondroplasia In Children

Ascendis Pharma

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(RTTNews) - Ascendis Pharma A/S (ASND) announced that the U.S. Food & Drug Administration (FDA) has granted accelerated approval for YUVIWEL (navepegritide; developed as TransCon CNP), the first and only once-weekly treatment indicated to increase linear growth in children aged two years and older with achondroplasia who have open epiphyses. YUVIWEL is also the only therapy to provide continuous systemic exposure to CNP over the weekly dosing interval. Continued approval for this indication, which was based on improvements in annualized growth velocity (AGV), may depend on verification and description of clinical benefit in confirmatory trials.

Achondroplasia is a rare genetic condition that causes skeletal dysplasia and can lead to muscular, neurological, and cardiorespiratory complications. YUVIWEL, a prodrug of C-type natriuretic peptide (CNP), is administered once weekly and designed to provide continuous exposure of active CNP to receptors throughout the body, counteracting the overactive FGFR3 signaling that drives achondroplasia.

The FDA's decision was based on its review of the clinical package for TransCon CNP submitted with Ascendis' New Drug Application. This included safety and efficacy data from three randomized, double-blind, placebo-controlled clinical trials, along with up to three years of open-label extension data. The pivotal ApproaCH Trial results were published in JAMA Pediatrics.

Ascendis expects to make YUVIWEL available to prescribing physicians in the United States in the early part of the second quarter of 2026. The company also plans to launch patient services through its Ascendis Signature Access Program (A.S.A.P.), offering support for treatment navigation and financial assistance programs for eligible patients.

With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV), which grants priority review to a subsequent drug application that would not otherwise qualify. This program is designed to encourage the development of new drugs and biologics for rare pediatric diseases.

ASND closed Friday's regular trading session at $233.50, reflecting a gain of $4.51 or 1.97%. In after-hours trading, the stock continued its upward momentum, rising to $243.00, an increase of $9.50 or +4.07%.

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