Ultragenyx Announces Plans to File for Accelerated Approval of UX111 for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)
Orphan DrugFast TrackGene TherapyClinical Study
Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval
Data from existing clinical studies appear sufficient for a biologics license application (BLA) submission
June 12, 2024 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company held a successful meeting with the U.S. Food and Drug Administration (FDA or the Agency) during which the company reached agreement with the Agency that cerebral spinal fluid (CSF) heparan sulfate (HS) is a reasonable surrogate endpoint that could support submission of a biologics license application (BLA) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy for the treatment of Sanfilippo syndrome (MPS IIIA). The company will need to finalize details of its BLA with the Agency in a pre-BLA meeting with the intent to file late this year or early next.
“Gaining alignment with the FDA that CSF HS is a relevant biomarker to enable accelerated approval in Sanfilippo syndrome is a pivotal moment for the community and paves the way for treatments for all fatal types of neuronopathic mucopolysaccharidoses,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “We commend the FDA for appreciating the critical urgency to deliver potentially life-saving therapies to children with neurologically devastating diseases and we extend our gratitude to the patient and caregiver advocates, scientists and industry leaders that shared and collaborated to provide the comprehensive evidence needed to support this important decision.”
As discussed with the FDA, the BLA filing will be based on the available data including from the ongoing pivotal Transpher A study evaluating the safety and efficacy of UX111 in children with MPS IIIA. Results from the Transpher A and long-term follow-up studies were recently presented at the 20th Annual WORLDSymposium™.
About UX111
UX111 is a novel in vivo gene therapy in Phase 1/2/3 development for Sanfilippo syndrome type A (MPS IIIA), a rare fatal lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). UX111 is designed to be dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of heparan sulfate, a glycosaminoglycan, in the brain that results in progressive cell damage and neurodegeneration. The UX111 program has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the U.S., and PRIME and Orphan medicinal product designations in the EU.
About Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodegeneration, with onset in early childhood. Children with MPS IIIA present with global developmental delay which eventually leads to progressive language and cognitive decline, behavioral abnormalities and early death. MPS IIIA is estimated to affect approximately 3,000 to 5,000 patients in commercially accessible geographies with a median life expectancy of 15 years MPS IIIA is caused by biallelic pathogenic variants in the SGSH gene that lead to a deficiency in the sulfamidase (SGSH) enzyme responsible for breaking down heparan sulfate, a glycosaminoglycans, which accumulate in cells throughout the body resulting in the observed rapid neurodegeneration that is associated with the disorder.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
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