Health
Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA)
Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotype BLA resubmitted to U.S. FDA in January 2026; Company expects up to six-month review period per FDA guidelines NOVATO, Calif., Feb. 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. today announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lyso
About this update from Ultragenyx Pharmaceutical Inc.
[{"type":"image","alt":"Ultragenyx Pharmaceutical Inc.","displaySize":"","headline":null,"caption":"Ultragenyx Pharmaceutical Inc.","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":113,"url":"https://media.zenfs.com/en/globenewswire.com/98c2a760a19e68caceb0f8447079e784"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/WFxQplTkxt9FOMPM4ARqBw--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTE1ODtjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/98c2a760a19e68caceb0f8447079e784","width":300,"height":113}},"lazy":false},{"type":"text","content":"Data represents up to 8.5 years of follow-up and are consistent across age, dose, and genotype","length":94,"tagName":"p"},{"type":"text","content":"BLA resubmitted to U.S. FDA in January 2026; Company expects up to six-month review period per FDA guidelines ","length":109,"tagName":"p"},{"type":"text","content":"NOVATO, Calif., Feb. 03, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. today announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec), an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder. The results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with natural history, with consistent and highly statistically significant results across age and disease severity. UX111 was well-tolerated and the safety profile remains favorable.","length":616,"tagName":"p"},{"type":"text","content":"“These data continue to demonstrate a remarkable and unprecedented separation from the natural history of Sanfilippo syndrome through more than eight years of follow-up, with children in their teens retaining skills at an age when many of their untreated peers have sadly lost their most basic abilities and succumbed to this disease,” Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “Our studies consistently show that reductions in heparan sulfate are associated with meaningful clinical benefits across multiple domains, underscoring the urgency to bring forward a treatment for families who currently have no options to stop or delay the heartbreaking and inevitable progression and loss of function associated with this disease.”","length":768,"tagName":"p"},{"type":"text","content":"The dat...