Biomarin has granted Ascendis a non-exclusive, worldwide license to use navepegritide, the active ingredient in the latter company’s recently approved achondroplasia drug Yuviwel, in exchange for 18–20% royalties on net sales.
Biomarin and Ascendis Pharma have settled a legal battle over the patents for the companies’ rival achondroplasia drugs. Analysts heralded the agreement as a win for Biomarin, which scored a higher-than-expected royalty rate on sales of Ascendis’ Yuviwel.
At issue is the European patent for Biomarin’s Voxzogo. The company argued in its January 2025 complaint that Ascendis’ therapy, which at the time was under development as TransCon CNP, infringed on the patent for Voxzogo. The patent that had been awarded for Voxzogo covered long-acting variants of C-type natriuretic peptide (CNP).
TransCon CNP, which was approved in February as Yuviwel, is a pro-drug version of CNP that is designed to increase exposure of the active ingredient and improve efficacy and safety.
The case eventually came to center on legal hang ups around Ascendis’ attempt to delay hearing the case while Yuviwel was under review by the FDA. The court ruled against Ascendis in March, shortly after it secured approval for Yuviwel in February.
But the underlying patent issues had yet to be resolved. Now, the two sides have come to an agreement, according to separate press releases issued Monday by the companies.
Biomarin has granted Ascendis a non-exclusive, worldwide license to use Yuviwel’s active ingredient, which is called navepegritide, in achondroplasia, hypochondroplasia and other indications.
“We view the agreement as a win-win for Ascendis and BioMarin,” BMO Capital Markets wrote in a Monday morning note.
Ascendis will pay 18–20% royalties on net sales to Biomarin, depending on where the drug is sold, through May 20, 2030.
“The royalty rates appear relatively high compared with recent biotech patent settlements, while the duration is relatively short,” BMO said.
In a separate note to investors, Stifel said a settlement had been expected but not the high royalty rate. “For legal cases there’s never really a perfect precedent or analog, but a high-teens/20% royalty is at the high end of these types of agreements,” the firm said.
Analysts believe the timeline matches up with Biomarin’s plan to launch the investigational BMN 333 as a once-weekly, long-acting CNP option for the diseases.
Ascendis can now move forward unimpeded with Yuviwel, a drug that BMO believes has wide potential “to lead the achondroplasia and broader CNP market.”
“The ongoing rapid uptake of Yuviwel in the United States, we believe, reflects its differentiated profile and the large, unmet medical need it is addressing as an important new foundation of care in achondroplasia and other types of skeletal dysplasia,” said Jan Møller Mikkelsen, CEO of Ascendis, in a Monday statement.
Biomarin, on the other hand, is facing a loss of market share for Voxzogo but will now get a nice chunk of change from its rival out of the settlement.
“For BioMarin, the high royalties provide meaningful participation in Yuviwel’s growth and should partially offset expected Voxzogo erosion as the weekly competitor gains adoption,” BMO said. Now, Biomarin can focus on BMN 333, the firm continued.
Stifel said that the agreement “signifies confidence” in potential next-gen drug BMN 333. Preliminary data from a Phase 3 trial of the asset is due in 2027.
“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,” said Alexander Hardy, CEO of BioMarin, in a prepared statement.
