Ultragenyx announces plans to file for accelerated approval of UX 111,gene therapy, for the treatment of Sanfilippo syndrome Type A (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 UX 111 in children with MPS IIIA. Results from the Transpher A and long-term follow-up studies were recently presented at the 20th Annual WORLD Symposium™.