Recent Updates — RARE
Ultragenyx completed the sale of a Priority Review Voucher for $210 million, receiving gross proceeds upon closing. The company also announced a restructuring plan involving a 20% workforce reduction to reduce expenses, estimating approximately $25 million in total charges primarily related to severance and employee-related costs. These actions are expected to drive at least a 50% reduction in 2027 R&D expenses compared to 2025 levels. Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases.
Ultragenyx Pharmaceutical Inc., Baylor Research Institute, and Navinta LLC entered into a settlement agreement resolving patent litigation concerning the Company's drug DOJOLVI (triheptanoin). The dispute arose from Navinta's Abbreviated New Drug Application to market a generic version of the product. Under the terms, Ultragenyx grants Navinta a non-exclusive, royalty-free license to sell its generic DOJOLVI beginning January 1, 2033, potentially extending to July 1, 2033 if pediatric exclusivity is granted. Other pending litigation against different ANDA filers remains active. The company operates in the biopharmaceutical industry, developing treatments for rare genetic diseases.
Ultragenyx Pharmaceutical Inc. entered into an asset purchase agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) to a buyer for $203 million in cash. The company was awarded the PRV by the U.S. Food and Drug Administration on September 17, 2026, following the approval of FAYUVI (rebisufligene etisparvovec-hopf) for treating pediatric patients with mucopolysaccharidosis type IIIA. The transaction is subject to customary closing conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Ultragenyx Pharmaceutical Inc. operates in the biopharmaceutical industry, focusing on developing treatments for rare genetic diseases.
Ultragenyx Pharmaceutical Inc. entered into an asset purchase agreement to sell a Rare Pediatric Disease Priority Review Voucher (PRV) it received from the FDA in August 2026 upon approval of GENGLYCOS™. The buyer will pay $210 million in cash at closing, subject to customary conditions including Hart-Scott-Rodino antitrust review. Ultragenyx must remit 20% of gross proceeds to the U.S. Department of Health under a patent license agreement with the NIH. This biotechnology company develops treatments for rare genetic diseases.
On September 17, 2026, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration granted standard full approval for FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, to treat pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA). This marks the first FDA-approved treatment for Sanfilippo syndrome Type A and serves as the company's second gene therapy approval. Additionally, Ultragenyx received a Priority Review Voucher associated with this regulatory milestone. The company operates in the biopharmaceutical industry, focusing on developing treatments for rare genetic diseases.
Ultragenyx Pharmaceutical announced that its Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome failed to achieve the primary endpoint of change from baseline in Bayley-4 cognitive raw score or the key secondary endpoint of net response in the Multidomain Responder Index. The safety profile was consistent with earlier phases, but no efficacy differences supported approval. The company will evaluate the program's future and implement significant expense reductions while supporting its commercial business. Ultragenyx Pharmaceutical Inc. operates in the biopharmaceutical industry, developing treatments for rare genetic diseases.