BioMarin said on October 9, 2026 that it has submitted a supplemental application to the FDA for Voxzogo (vosoritide), a C-type natriuretic peptide analog approved for achondroplasia, in hypochondroplasia, a milder genetic form of short stature, with filings in Europe and other regions on track and a possible 2027 launch if approved. Phase 3 results showing faster growth in children with hypochondroplasia are among 13 studies the company is presenting at the ASBMR meeting in Boston, though the release gives no growth figures. A separate application with long-term achondroplasia data has an FDA decision date of February 28, 2027.
Ascendis Pharma (NASDAQ: ASND) and BioMarin Pharmaceutical (NASDAQ: BMRN) announced Monday August 31, 2026 a binding term sheet for a global settlement and license agreement resolving all litigation and disputes related to YUVIWEL (navepegritide, TransCon C-type natriuretic peptide for achondroplasia and hypochondroplasia). Terms: BioMarin grants Ascendis a non-exclusive, worldwide, royalty-bearing license to continue researching, developing, manufacturing, and commercializing navepegritide-related products without restriction, and waives certain regulatory rights and exclusivities. Ascendis makes royalty payments to BioMarin of 20% on U.S. net sales and 18% on European Union, South Korea, and Brazil net sales from first commercial sale through May 20, 2030. BioMarin dismisses the pending U.S. International Trade Commission Section 337 investigation plus litigation in Brazil, Denmark, Germany, South Korea, and the U.S. Northern District of California. The settlement covers all current and potential YUVIWEL indications, including achondroplasia and hypochondroplasia, and removes the largest overhang on the Ascendis TransCon peptide platform.