Ascendis surges on FDA approval of first once-weekly achondroplasia drug
YUVIWEL's accelerated approval marks competitive milestone against BioMarin's daily treatment in rare disease market
Ascendis Pharma shares climbed 2% to $233.50 on Friday, approaching the stock's 52-week high of $242, after the U.S. Food and Drug Administration granted accelerated approval for YUVIWEL (navepegritide) as the first and only once-weekly treatment for children with achondroplasia.
The Danish biotechnology company secured the regulatory milestone for patients aged two years and older with open epiphyses, with commercial availability expected in the second quarter of 2026. Ascendis also received a Rare Pediatric Disease Priority Review Voucher alongside the approval, a valuable asset that can be sold to other drugmakers or used to accelerate future regulatory submissions.
"This approval represents a transformative moment for children with achondroplasia and their families," the company stated in the FDA announcement. "YUVIWEL is the first therapy to provide continuous systemic exposure to CNP over a weekly dosing interval."
The achondroplasia treatment market, while niche, is expanding rapidly as targeted therapies emerge. Industry estimates place the current market between $137 million and $300 million, with projections reaching $655 million to $1.5 billion by the mid-2030s. The variability in forecasts reflects the dynamic nature of this specialized therapeutic area, which is shifting from conventional symptomatic management to targeted biological approaches.
YUVIWEL enters a market currently dominated by BioMarin's Voxzogo (vosoritide), a daily injection that generated $927 million in 2025 sales, representing 26% year-over-year growth. BioMarin projects Voxzogo revenue between $975 million and $1.025 billion for 2026, with approximately 5,000 children globally receiving treatment as of year-end 2025.
The once-weekly dosing regimen of YUVIWEL could prove a significant competitive advantage, potentially improving adherence and convenience for patients and caregivers. Analysts at Royal Bank of Canada highlighted this distinction, noting the therapy's potential to capture market share in the growing achondroplasia treatment landscape.
The approval comes after a three-month delay from the original Prescription Drug User Fee Act target date of November 30, 2025. The FDA extended the review timeline to February 28, 2026, to review updated materials related to post-marketing requirements, which the agency classified as a major amendment to the New Drug Application.
Analysts maintain an overwhelmingly positive outlook on Ascendis, with an average rating of "Buy" or "Strong Buy" and consensus price targets ranging from $276 to $290. Morgan Stanley reiterated an "overweight" rating with a $256 target on February 12, while Barclays set an even more bullish target of $342 in late January. Royal Bank of Canada increased its price objective from $245 to $250 in January, and Bank of America boosted its target from $246 to $260 on January 30.
The FDA approval caps a period of strong commercial execution for Ascendis. The company reported fourth-quarter 2025 revenue of €248 million, up from €174 million in the prior-year period, driven primarily by YORVIPATH, which generated €187 million in quarterly sales. Full-year revenue more than doubled to €720 million from €364 million in 2024, though the company recorded a net loss of €228 million amid heavy investment in commercial infrastructure.
"Ascendis is transitioning into a revenue-scaled commercial biotech, largely driven by the rapid adoption of YORVIPATH," analysts noted following the earnings release. The company achieved operating profit of €10 million in Q4 2025 and generated €73 million in operating cash flow, suggesting its core business can self-fund growth.
Looking ahead, Ascendis has submitted a marketing authorization application to the European Medicines Agency, with a regulatory decision anticipated in the fourth quarter of 2026. The company is also conducting a label expansion trial for infants with achondroplasia, with enrollment expected to complete in 2026, and is exploring a combination therapy pairing TransCon CNP with TransCon Growth Hormone.
Competition in the achondroplasia space is intensifying. BridgeBio Pharma is developing an oral FGFR inhibitor, infigratinib, which demonstrated positive Phase 3 data and could launch as early as 2027. BioMarin is also advancing BMN 333, a next-generation achondroplasia therapy with pivotal studies planned for 2026 and potential launch in 2030.
With a market capitalization of approximately $14 billion and trading at 19.4 times trailing sales, investors are pricing in significant expectations for YUVIWEL's commercial success. The stock has rallied from its 52-week low of $124.06, reflecting growing confidence in Ascendis's transition from clinical-stage biotechnology to commercial enterprise with multiple revenue streams.