Also known as: Dojolvi
Dojolvi is a triheptanoin oral therapy approved in the U.S. for long-chain fatty acid oxidation disorders (LC-FAOD) across adult and pediatric populations. It is described as the first and only FDA-approved treatment for LC-FAOD and serves as a source of calories and fatty acids. FDA marketing approval date is June 30, 2020.
Ultragenyx began a restructuring plan that includes a 10% workforce reduction and termination of UX143 manufacturing agreements. The company also faces a class action tied to setrusumab disclosures.
Opus Genetics reported positive early data from its BEST1 gene therapy program and expects FDA action on its presbyopia treatment in October 2026. Ultragenyx announced 2025 revenue of $673 million and initiated a strategic restructuring plan targeting profitability in 2027.
Ultragenyx Pharmaceutical received an Incomplete Response Letter from the FDA for its UX111 gene therapy resubmission for Sanfilippo syndrome type A, requesting additional documentation. The company announced a 10% workforce reduction and expects profitability by 2027.
Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.
