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.
"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.
"The data supported durable clinical benefit," Barclays analyst Geena Wang said in a note to clients at the time. Wang added she was "optimistic on the FDA's openness and flexibility for accelerated approval paths in rare disease" based on prior agency comments.