Rebisufligene etisparvovec

Ultragenyx submitted a BLA to the FDA for UX111, a gene therapy for Sanfilippo Syndrome Type A, marking a potential first approved treatment in the U.S. UX111 targets the underlying enzyme deficiency, showing promise in reducing disease biomarkers and improving cognitive development.

Ultragenyx Pharmaceutical Inc. submitted a BLA to the FDA for UX111, a gene therapy for Sanfilippo syndrome type A, marking a potential first approved treatment in the U.S. UX111 targets the underlying SGSH enzyme deficiency, showing promise in reducing heparan sulfate levels and improving cognitive development.

Ultragenyx Pharmaceutical Inc. submitted a BLA to the FDA for accelerated approval of UX111, a gene therapy for Sanfilippo Syndrome Type A, marking a potential first U.S. therapy for the condition. UX111 aims to address the underlying enzyme deficiency causing the disease, with data showing reduced HS levels in CSF and improved cognitive development. The therapy has received multiple designations for expedited review.

Ultragenyx (RARE) submitted a BLA to the FDA for accelerated approval of UX111, ABO-102, an AAV gene therapy for Sanfilippo syndrome type A treatment.

Ultragenyx reported Q3 2024 revenue of $139 million, up 42% YoY, with Crysvita® at $98 million and Dojolvi® at $21 million. The company reaffirmed 2024 revenue guidance of $530-$550 million and announced Breakthrough Designation for setrusumab (UX143) in osteogenesis imperfecta. DTX401 Phase 3 data showed a 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa).

Sanfilippo syndrome type A (MPS IIIA) is a fatal CNS disorder caused by heparan sulfate accumulation due to SGSH gene mutations, leading to rapid neurodegeneration. UX111, an investigational gene therapy, aims to address the enzyme deficiency causing this accumulation, with ongoing clinical trials and regulatory designations.

Ultragenyx plans to submit a protocol amendment for the phase 1/2/3 Cyprus2+ clinical trial evaluating UX701, an AAV vector-based gene therapy for Wilson disease, to include a higher dose cohort and optimized immunomodulation regimen. The new cohort will be added to stage 1, which has already dosed 15 patients. Six patients have fully tapered off standard of care treatment, with stabilization of nonceruloplasmin bound copper levels and improved ATP7b gene function noted. The gene therapy has been well-tolerated with no significant safety events.