Ultragenyx preps US filing for Sanfilippo syndrome gene therapy
Gene TherapyClinical ResultPhase 2
Ultragenyx Pharmaceutical announced plans to file for accelerated approval of its gene therapy UX111 for the rare, fatal lysosomal storage disease Sanfilippo syndrome type A (MPS IIIA). The decision follows the FDA agreeing that levels of the biomarker heparan sulfate in cerebral spinal fluid (CSF-HS) can serve as a reasonable surrogate endpoint to support the application.
The biotech stated it will finalise details of the planned US regulatory filing with the FDA, and aims to submit before the end of 2024 or early 2025. It acquired exclusive global rights to the investigational AAV gene therapy, formerly known as ABO-102, through a $30-million deal with Abeona Therapeutics in 2022.
Alignment with FDA
"Gaining alignment with the FDA that CSF heparan sulfate 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 Ultragenyx CEO Emil Kakkis.
The announcement follows clinical data updates presented this past February from the pivotal TRANSPHER A study evaluating the one-time intravenous infusion. As of an August 2023 data cutoff, treatment with UX111 led to rapid and sustained reductions in CSF-HS that correlated with stabilisation or gains in cognitive function across 17 modified intent-to-treat patients.
Meanwhile, Ultragenyx is also preparing regulatory submissions for its DTX401 gene therapy in glycogen storage disease type 1a next year and recently reported longer-term Phase II data for its anti-sclerostin antibody setrusumab, partnered with Mereo BioPharma, in the rare inherited brittle bone disease osteogenesis imperfecta.
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