Health
Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA
Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be presented at WORLDSymposium™ 2026 NOVATO, Calif., Jan. 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has resubmitted its Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a trea
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":"Company expects up to six-month review period per FDA guidelines ","length":64,"tagName":"p"},{"type":"text","content":"New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be presented at WORLDSymposium™ 2026","length":163,"tagName":"p"},{"type":"text","content":"NOVATO, Calif., Jan. 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has resubmitted its Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) to the U.S. Food and Drug Administration (FDA or the Agency). The submission contains substantial longer-term data on multiple measures of neurologic benefit to support an intermediate clinical endpoint for accelerated approval supported further by CSF heparan sulfate and other biomarker data, as agreed with the FDA during the last clinical review.","length":683,"tagName":"p"},{"type":"text","content":"“Today, with no approved treatment options to address the relentless progression of Sanfilippo syndrome type A, families must watch helplessly as their children lose the ability to communicate, play, move, and even eat before ultimately succumbing to this devastating and fatal disease,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We recognize the extraordinary stakes facing the Sanfilippo community as they await a first-ever treatment option and look forward to working with the Agency as it completes its review of this urgently needed therapy. There is no time to waste, and we believe we have addressed all of the Agency’s concerns to avoid further delays. Patients, and their familie...