Mepsevii is the brand name for vestronidase alfa, a recombinant human lysosomal beta-glucuronidase enzyme replacement therapy. It is indicated for mucopolysaccharidosis type VII (Sly syndrome). The FDA first approved Mepsevii on November 15, 2017.
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 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.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT03775174 |
Expanded Access to Mepsevii |
AVAILABLE |
