The US Food and Drug Administration (FDA) has granted accelerated approval to Ascendis Pharma’s Yuviwel (navepegritide) for the treatment of a rare genetic condition in children that causes dwarfism.
The once-weekly therapy will be available to children aged two and over with achondroplasia with open growth plates. The FDA’s accelerated approval is for the increase of linear growth in these patients. Ascendis expects to make its drug available through prescribing physicians in the US during Q2 2026.
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The FDA based its decision on three randomised, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data. In the Phase III ApproaCH study (NCT05598320), treatment with Yuviwel, at the time called TransCon CNP, led to significantly higher annualised growth velocity (AGV) at week 52 compared to placebo, meeting the trial’s primary endpoint. Data also showed improved lower-limb alignment and body proportionality and positive changes in health-related quality of life (QoL), with safety similar to placebo. Ascendis said that continued approval for this indication may be contingent upon verification and description of clinical benefit in one or more confirmatory trials.
In November, the FDA delayed its decision on Ascendis’ drug by three months to give itself more time to review post-marketing requirement data. The drug was originally accepted on the agency’s priority review pathway. With accelerated approval now secured, the FDA has issued Ascendis a rare paediatric disease priority review voucher (PRV). Ascendis may choose to use it on another drug’s application from its pipeline or sell it to another pharma company – the current price they fetch is around $200m.
Achondroplasia is a rare genetic condition causing skeletal dysplasia. It is the most common form of dysplasia, known as short-limbed dwarfism. Patients also have an increased risk of muscular, neurological, and cardiorespiratory complications. While usually a spontaneous mutation, it can be inherited as an autosomal dominant trait.
Yuviwel is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signalling in achondroplasia.
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By GlobalDataBioMarin’s Voxzogo (vosoritide) won FDA approval in paediatric achondroplasia in November 2021 and secured a label extension in October 2023, but this therapy is a daily injection, while Yuviwel is a subcutaneous injection administered once weekly.
Carlos Bacino, professor of molecular and human genetics at Baylor College of Medicine and Texas Children’s Hospital, and also investigator on the ApproaCH trial, said: “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.”
The approval comes soon after the pharma industry commemorated rare disease day. Over the past year, the FDA has shifted policies and guidance to improve innovation in the space. This includes the most plausible mechanism pathway and clinical trial reforms, such as sponsors being able to use flexible study designs.
Moreover, the rare paediatric PRV scheme, which Ascendis has benefited from in Yuviwel’s approval, was reauthorised in February 2026. Rare disease charities welcomed the framework’s return to the pharma landscape.
