Regulatory & Policy

BioMarin lands 20% Yuviwel royalty in Ascendis patent settlement

BioMarin lands 20% Yuviwel royalty in Ascendis patent settlement

A patent dispute that spanned multiple continents and two of the most closely watched rare disease drugs has ended with BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) securing a royalty-bearing settlement from Denmark-based Ascendis Pharma A/S (Nasdaq: ASND) on sales of navepegritide (Yuviwel), the once-weekly C-type natriuretic peptide (CNP) analog that received FDA accelerated approval in February 2026 for achondroplasia in children aged two and older. Under the binding terms announced August 30, Ascendis will pay BioMarin 20% of Yuviwel net sales in the US, retroactive to the first commercial sale, and 18% of net sales in the EU, Brazil, and South Korea through May 2030. The agreement resolves all pending proceedings between the companies. The license covers all current and potential indications for Yuviwel, including achondroplasia and hypochondroplasia.

BioMarin now collects a royalty stream from a commercially launched competitor product — an outcome that extracts significant value from its C-type natriuretic peptide patent estate. Ascendis reported Yuviwel revenue of EUR 8 million (approximately USD 8.8 million) in Q2 2026 alone, its first full quarter following the April 2026 commercial launch.

The dispute centered on BioMarin patents covering CNP analogs and their use in skeletal dysplasias driven by FGFR3 gain-of-function mutations. Ascendis' TransCon platform conjugates a CNP analog to an inert carrier through a hydrolyzable linker, enabling once-weekly dosing compared with the daily subcutaneous administration of vosoritide. By taking a license covering Yuviwel across current and potential indications, Ascendis removes the patent uncertainty surrounding its competing CNP franchise without disrupting commercialization.

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Both products currently hold accelerated approval in the US, with confirmatory trial obligations pending. BioMarin's supplemental New Drug Application for full traditional approval of Voxzogo was accepted by FDA in July 2026, with a PDUFA target date of February 28, 2027. On the indication expansion front, BioMarin reported positive Phase III results for vosoritide in hypochondroplasia in May 2026, with an annualized growth velocity improvement of 2.33 cm/year versus placebo (p<0.0001) and a supplemental NDA submission planned for Q3 2026. The settlement license explicitly covers hypochondroplasia, meaning any future Yuviwel approval in that indication would also carry royalty obligations to BioMarin.

The most significant competitive variable outside the CNP class is infigratinib, an oral FGFR3 kinase inhibitor developed by BridgeBio Pharma and QED Therapeutics. Unlike Yuviwel, infigratinib acts intracellularly on FGFR3 and sits outside BioMarin's CNP patent estate, meaning a future approval would introduce a competitor from which BioMarin would not benefit through the Ascendis settlement. The drug reported positive Phase III results, with an NDA filing planned for H2 2026.


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