Also known as: Evkeeza
Evkeeza (evinacumab-dgnb) is an FDA-approved monoclonal antibody therapy used as an adjunct to standard lipid-lowering management for homozygous familial hypercholesterolemia (HoFH). It was initially approved in 2021 and has since expanded to younger pediatric patients, with current labeling including patients aged 1 year and older. The drug is given as monthly intravenous infusion.
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.
