Vosoritide

Agreement Resolves all Pending Patent-Related Proceedings Between BioMarin and Ascendis SAN RAFAEL, Calif., Aug. 30, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has entered into binding terms with Ascendis Pharma A/S, resolving the patent and ancillary disputes pending globally, including before the U.S. International Trade Commission (ITC) concerning Ascendis's Yuviwel. As part of the agreement, Ascendis will pay BioMarin a royalty equal to 20% of net sales of Yuviwel in the U.S., retroactive to the first commercial sale, and 18% of net sales in the European Union, Brazil and South Korea until May 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," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We have spent decades focused on understanding the underlying biology of rare genetic conditions, building the deep scientific expertise that led to our development of six first-in-disease medicines for patients. We look forward to continuing to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on our ongoing momentum for children with achondroplasia." The scope of the settlement includes a license for BioMarin's patents that relate to Yuviwel for all current and potential indications, including achondroplasia and hypochondroplasia. It also covers the use of Yuviwel in combination with other medicines. Under the terms of the agreement, BioMarin will dismiss the pending Section 337 investigation before the ITC and the parties will resolve all claims relating to the asserted intellectual property, including litigation pending in Brazil, Denmark, Germany, South Korea and the Northern District of California. Reaching this agreement recognizes the value of BioMarin's pioneering innovations in C-type natriuretic peptide (CNP) technology, including the development of VOXZOGO® (vosoritide), while providing a framework that enables continued access to medicine for children with achondroplasia around the world. About BioMarin BioMarin is a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. Founded in 1997, the San Rafael, California-based company has a proven track record of innovation, with nine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, BioMarin seeks to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients. To learn more, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the settlement and license agreement with Ascendis Pharma A/S, including expected benefits of such agreement and anticipated royalty payments, and future commercialization of licensed products and BioMarin's expectations to continue to innovate, bringing forward the next generation of medicines for people with serious genetic conditions, and building on its momentum for children with achondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's ability to enforce the agreement; actual sales of licensed products; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as such factors may be updated by any subsequent reports. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. SOURCE BioMarin Pharmaceutical Inc. 21% more press release views with Request a Demo

iStock, maystra 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. Approvals Ascendis Enters Achondroplasia Arena as FDA Approves First Weekly Drug Yuviwel will compete with BioMarin’s Voxzogo. Meanwhile, BridgeBio is working to bring its own achondroplasia drug, the FGFR3 blocker infigratinib, to the market. March 2, 2026 · 2 min read · Tristan Manalac Read more 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. Rare diseases BioMarin bounces back as Voxzogo performs ‘bigger than expected’ in new indication After being hit by safety issues and subpar results in another trial, BioMarin’s Phase 3 test of Voxzogo for a rare skeletal disorder called hypochondroplasia showed efficacy “solidly above” what the drug has shown for achondroplasia, which causes dwarfism. May 21, 2026 · 2 min read · Tristan Manalac Read more 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 pro 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 .