Yuviwel will compete with BioMarin’s Voxzogo. Meanwhile, BridgeBio is working to bring its own achondroplasia drug, the FGFR3 blocker infigratinib, to the market.
The FDA has given the go-ahead to Ascendis Pharma’s navepegritide to boost growth in kids 2 years and older with achondroplasia. The drug will be marketed under the brand name Yuviwel.
Yuviwel is the first drug on the market to use weekly dosing to provide sustained systemic exposure to C-type natriuretic peptide (CNP), a key signaling molecule affected in achondroplasia, according to Ascendis’ Friday news release. Yuviwel will be available to patients through prescribing physicians in the “early part of the second quarter,” the biotech said.
Achondroplasia is a genetic disease characterized by faulty bone development in children. Though classified as a rare disease, achondroplasia is the most common cause of dwarfism. Navepegritide, Yuviwel’s active ingredient, addresses a key disease pathway by providing continuous exposure to CNP, thus promoting bone growth.
Pivotal findings from the Phase 2b APPROACH study showed that annualized growth velocity increased by 1.49 cm/year in patients on Yuviwel versus placebo—a treatment effect that was statistically significant—as per data published in JAMA Pediatrics in November 2025. Yuviwel’s edge over placebo started 12 weeks after treatment initiation and persisted throughout the study.
Yuviwel also improved the ratio in children’s upper-to-lower body segments, indicating more proportional bone growth.
Alongside Friday’s approval, Ascendis also received a rare pediatric disease priority review voucher from the FDA, which it can use to secure an expedited review of a drug that would have otherwise gone through the agency’s standard timeline. The biotech also has the option to sell this voucher, which could go for hundreds of millions of dollars.
Yuviwel could “transform the treatment of achondroplasia,” Ascendis CEO Jan Mikkelsen said in a statement.
Currently, the only other available treatment for achondroplasia is BioMarin’s Voxzogo, which similarly targets the CNP disease pathway but is injected daily, as opposed to Yuviwel’s weekly schedule. The drug was approved in November 2021 and seems to be waning in popularity. During the company’s third-quarter 2025 earnings call, BioMarin walked back its previous goal of hitting $4 billion revenue by 2027, a decision that CEO Alexander Hardy said at the time was heavily influenced by incoming competition to Voxzogo.
Besides the two marketed therapies from Ascendis and BioMarin, other companies are advancing their own achondroplasia therapies. These include BridgeBio and its FGFR3 blocker infigratinib, which is currently in late-stage development. Data from the Phase 3 PROPEL 3 study, released last month, showed an annualized height velocity improvement of 2.10 cm/year versus placebo. A new drug application is slated for the second half of 2026.
