Ultragenyx Pharmaceutical has successfully convinced the U.S. Food and Drug Administration (FDA) to recognize a biomarker as a surrogate endpoint, allowing the company to seek accelerated approval for its gene therapy for Sanfilippo syndrome. This pivotal decision marks a significant step forward for Ultragenyx, which plans to file for approval of its UX111 AAV gene therapy by the end of the year.
The journey began when Ultragenyx acquired global rights to UX111 from Abeona Therapeutics in 2022. The pivotal trial for this gene therapy was already in progress. Despite early data showing positive results, Ultragenyx faced uncertainty over whether the FDA would accept cerebral spinal fluid (CSF) heparan sulfate (HS) as a basis for approval. The company presented its case at a Reagan-Udall Foundation event, attended by FDA staff, to argue for the biomarker’s acceptance.
Ultragenyx announced on Wednesday that it had reached an agreement with the FDA, which now recognizes CSF HS as a reasonable surrogate endpoint for accelerated approval. This agreement allows Ultragenyx to finalize its filing details and seek FDA approval either late in 2024 or early in 2025.
Ultragenyx Prepares FDA Filing for Gene Therapy Following Promising Sanfilippo Syndrome Study Results
The filing will be based on data from the ongoing pivotal study of UX111. Earlier this year, Ultragenyx reported that CSF HS levels fell within one month of treatment in all 17 patients with Sanfilippo syndrome, a rare and fatal lysosomal storage disease. The company also observed improvements in cognitive function in 16 of the 17 patients, showing either stability or gains.
The forthcoming filing will reveal if the FDA considers the biomarker results and clinical efficacy sufficient for accelerated approval. Regardless, this approval to file is a milestone in Ultragenyx’s broader strategy to expand its portfolio of approved drugs swiftly. Ultragenyx also plans to seek approval for its DTX401 gene therapy for glycogen storage disease type Ia next year and anticipates phase 3 data for its brittle bone disease drug, setrusumab.
Bringing UX111 to market will increase Ultragenyx’s expenditures. The company has agreed to pay Abeona up to $30 million in commercial milestones following regulatory approval and tiered royalties of up to 10% on net sales. Ultragenyx began a public offering of up to $350 million of its common stock after this update on UX111.
Highlighting FDA Approval of Biomarker for Sanfilippo Syndrome Therapy
In a press release, Ultragenyx CEO Emil D. Kakkis emphasized the importance of the FDA’s recognition of CSF HS as a biomarker for accelerated approval. He praised the collaborative effort of patient advocates, scientists, and industry leaders in providing the necessary evidence for this critical decision. Kakkis highlighted the urgency of delivering potentially life-saving therapies to children with neurologically devastating diseases like Sanfilippo syndrome.
The UX111 gene therapy, designed to be administered via a one-time intravenous infusion, uses a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells. This therapy addresses the SGSH enzyme deficiency responsible for the accumulation of heparan sulfate in the brain, which leads to progressive cell damage and neurodegeneration. UX111 has received multiple designations in the US and EU, including Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug.
With this FDA agreement, Ultragenyx is poised to make significant strides in treating Sanfilippo syndrome and other severe genetic disorders, aligning with its mission to provide innovative therapies for rare and devastating diseases.
Reource: Ultragenyx Pharmaceutical, June 12, 2024
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