Regenxbio Successful Pre-BLA Meeting with FDA on Accelerated Approval in Mucopolysaccharidosis

 

• Aligned with FDA on content of BLA and plans for submission:
  • Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024
  • Confirmatory trial expected to begin in H2 2025
  • FDA confirmed RGX-121 commercial bulk drug is comparable to clinical material
• Positive biomarker, neurocognitive and systemic data will be part of BLA submission

https://www.prnewswire.com/news-releases/regenxbio-announces-successful-pre-bla-meeting-with-fda-to-support-accelerated-approval-pathway-for-rgx-121-for-the-treatment-of-mps-ii-302175172.html