Vosoritide

In a deal that will end intellectual property claims in five countries, including one before the International Trade Commission, Ascendis has agreed to pay a sizeable royalty to California-based BioMarin. The deal centers on Yuviwel, Acendis’ once-weekly injection for children with achondroplasia, a leading cause of dwarfism. A C-type natriuretic peptide (CNP) analog prodrug using transient conjugation drug delivery, Yuviwel received FDA accelerated approval in February and is in direct competition with BioMarin’s Voxzogo, a daily injection for achondroplasia approved in 2021. BioMarin alleged that Yuviwel violates its patent on CNP variants, thereby launching intellectual property disputes in the U.S., Brazil, Germany, South Korea and Denmark. Today the two pharmas sidestepped significant legal overhang by settling the dispute with a licensing deal that analysts are calling mutually beneficial. Per the terms, BioMarin now collects royalties on the more convenient competitor product. Ascendis will pay 20% of Yuviwel net sales in the U.S. dating back to the first commercial sales through to May 2030. Pending approval, Ascendis will also pay 18% of net sales in the European Union, Brazil and South Korea through May 2030. For BioMarin, consensus estimates cited by Jefferies have projected the royalty stream to be worth $280 million in U.S. revenue and $440 million in non-U.S. revenue by 2030. “This outcome incentivizes companies like BioMarin to keep investing in the kind of long-term innovation that is critical to bringing breakthrough treatments to the people who need them,” BioMarin CEO Alexander Hardy said in an Aug. 31 statement. In exchange, BioMarin will dismiss pending lawsuits and grant Ascendis a wide license to Yuviwel-related patents for both current and potential indications, allowing Ascendis to continue developing the CNP franchise without restriction. “The agreement allows BioMarin to participate economically in Yuviwel's commercial success, with royalties that should partly offset future Voxzogo sales erosion as patients switch to Ascendis' weekly therapy,” William Blair analysts said in a report. Ascendis reported 8 million euros ($9.3 million) in second-quarter revenue for Yuviwel after the drug’s April launch. In July, BioMarin said 90% of its U.S. patient base was still on Voxzogo, but that share is expected to shift to Yuviwel over time, William Blair noted. Meanwhile, Citi analysts called the agreement “net favorable” for the Yuviwel franchise. The royalties will cost Ascendis, but the deal removes the shroud of legal disputes and, perhaps more importantly, clears the way for future development in other skeletal dysplasia indications and in combination therapies currently under investigation at Ascendis, the Citi team argued. The settlement also specifically covers hypochondroplasia, a milder genetic bone disorder that causes shortened stature. BioMarin announced positive phase 3 results for Voxzogo in the indication in May 2026, and plans to file for supplemental approval by the end of the year. Ascendis has plans to start a similar phase 3 in hypochondroplasia, and any future approval would carry the same royalties through the 2030 window. “Importantly, the settlement has no bearing on the forward development of the TransCon CNP + hGH combo regimen (anticipated launch in 2030), which we continue to believe has best-in-class potential in [achondroplasia / hypochondroplasia],” Citi analysts wrote. In a late-summer readout of Ascendis’ phase 2 Coach trial, the combination therapy demonstrated durable and unprecedented growth in children with achondroplasia, matching the annualized growth velocity of the 97th percentile among children of average stature. The trial will complete around year-end 2026.