REGENXBIO Announces Successful Pre-BLA Meeting with FDA to Support Accelerated Approval Pathway for RGX-121 for the Treatment of MPS II

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 The FDA continues to be aligned with REGENXBIO's plan to use cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of br ...