Ascendis Ends Patent Dispute, Will Pay BioMarin 20% Royalty

Ascendis will pay BioMarin a royalty equal to 20% of Yuviwel sales in exchange for ending the patent dispute.

According to U.S. pharmaceutical trade publication Fierce Pharma on Aug. 31 (local time), Ascendis agreed to pay BioMarin a substantial royalty as part of a settlement ending intellectual property disputes in 5 countries, including the United States.

The settlement centers on Ascendis’ once-weekly injectable Yuviwel. The treatment is intended for children with achondroplasia, a major cause of dwarfism, and received accelerated approval from the U.S. Food and Drug Administration (FDA) in February.

BioMarin claimed that Yuviwel infringed its patent covering a C-type natriuretic peptide (CNP) variant and filed disputes in the United States, Brazil, Germany, South Korea and Denmark.

Under the settlement, Ascendis will pay BioMarin 20% of Yuviwel’s net sales in the United States from the first commercial sale through May 2030. If approved, it will also pay 18% of net sales in the European Union (EU), Brazil and South Korea through May 2030.

In return, BioMarin will withdraw the pending lawsuits and grant Ascendis a broad license to Yuviwel-related patents covering current and potential indications.

Alexander Hardy, BioMarin’s chief executive officer (CEO), said, “This outcome incentivizes companies like BioMarin to continue investing in the long-term innovation that is essential to delivering breakthrough treatments to the people who need them.”

According to market projections cited by Jefferies, the royalty revenue is estimated to reach $280 million in the United States and $440 million outside the United States by 2030.

William Blair analysts said, “The settlement allows BioMarin to participate economically in Yuviwel’s commercial success,” adding that “the royalty will partially offset the decline in Voxzogo revenue as patients switch to Ascendis’ once-weekly therapy.”

Ascendis reported €8 million (approximately 1.12 billion won) in Yuviwel revenue in the second quarter after its launch in April. BioMarin said in July that 90% of U.S. patients were still using Voxzogo, but William Blair expects that share to shift to Yuviwel over time.

Citi analysts said the settlement was “favorable for the Yuviwel franchise from a net income perspective.” They cited the removal of legal uncertainty despite the royalty burden and the opening of a path for Ascendis to develop other skeletal dysplasia indications and combination therapies under investigation.

The settlement also covers hypochondroplasia, a mild genetic bone disorder. BioMarin announced positive results in May from a phase 3 trial of Voxzogo in this indication and plans to seek an additional approval later this year.

Citi analysts said, “The settlement does not affect the future development of TransCon CNP and growth hormone combination therapy, which is expected to launch in 2030,” adding, “We continue to believe it has best-in-class potential in achondroplasia and hypochondroplasia.”