Ascendis, BioMarin Reach Global YUVIWEL License Deal

COPENHAGEN, Denmark, August 31, 2026

Global License Provides Continued Development Rights

Under the binding term sheet, Ascendis will retain broad rights to develop and commercialize navepegritide-related products globally. BioMarin has agreed to provide a covenant not to sue relating to the covered intellectual property and to dismiss all proceedings involving Ascendis. In return, Ascendis has agreed to dismiss its proceedings against BioMarin and make royalty payments based on net sales of navepegritide-related products. The royalty rate will be 20% of net sales in the United States and 18% in the European Union, South Korea and Brazil, beginning with the first commercial sale of YUVIWEL in the applicable country and continuing through May 20, 2030. The agreement is designed to provide greater clarity around the intellectual-property landscape while allowing Ascendis to continue expanding the commercial and development potential of YUVIWEL. The settlement comes as YUVIWEL gains commercial momentum in the United States. Ascendis previously reported that the product generated €8 million in second-quarter 2026 revenue and had more than 220 unique patient enrollments in the U.S. through July 31, with more than 65% of enrollments approved for reimbursement. The company has also indicated that it expects continued international expansion while regulatory review progresses in Europe.

YUVIWEL Targets Achondroplasia Growth

YUVIWEL (navepegritide) is a once-weekly therapy based on Ascendis Pharma’s TransCon CNP technology. Navepegritide is a prodrug of C-type natriuretic peptide (CNP) designed to provide continuous exposure to active CNP over the weekly dosing interval. Its mechanism is intended to counteract excessive FGFR3 signaling, an important driver of abnormal bone growth in achondroplasia. In February 2026, the U.S. Food and Drug Administration approved YUVIWEL to increase linear growth in pediatric patients aged 2 years and older with achondroplasia and open epiphyses. The therapy’s development has produced additional long-term clinical findings. Ascendis reported that Week 104 data from the pivotal ApproaCH trial demonstrated durable improvements in height, body proportionality and lower-limb alignment in children treated with TransCon CNP. The company also reported that treatment through two years was generally well tolerated, with a low rate of injection-site reactions and no symptomatic hypotension or acceleration of bone age observed in the reported data. These findings support continued investigation of navepegritide as a treatment approach for achondroplasia.

Settlement Supports YUVIWEL Expansion

The agreement could provide Ascendis with a clearer pathway for the continued global commercialization and development of YUVIWEL while resolving the legal disputes between the two pharmaceutical companies. Ascendis’ European Marketing Authorisation Application for YUVIWEL remains under review by the European Medicines Agency, with a regulatory decision anticipated in the fourth quarter of 2026. The company is also advancing additional clinical development programs, including the pivotal reACHin trial evaluating TransCon CNP in infants younger than two years with achondroplasia. Ascendis said the agreement reflects its continued focus on addressing the unmet medical needs associated with achondroplasia and other skeletal dysplasias. The company expects the settlement to allow research, development, manufacturing and commercialization activities involving navepegritide to continue without the restrictions associated with the previous disputes. The companies’ binding term sheet therefore represents both a significant intellectual-property resolution and an important commercial development for YUVIWEL as Ascendis works to expand access to the therapy across additional markets.

Source: Ascendis Pharma, BioMarin press release