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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
About this update from Regenxbio Inc.
[{"type":"text","content":"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 materialPositive biomarker, neurocognitive and systemic data will be part of BLA submissionROCKVILLE, Md., June 18, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it completed a successful Pre-Biologics License Application (BLA) meeting for RGX-121 for the treatment Mucopolysaccharidosis Type II (MPS II), where it finalized details of its BLA with the U.S. Food and Drug Administration (FDA).\nThe FDA continues to be aligned with REGENXBIO's plan to use cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of brain disease activity, as a surrogate endpoint reasonably likely to predict clinical benefit to support accelerated approval of RGX-121. Additionally, REGENXBIO and the FDA discussed manufacturing, non-clinical, device delivery system and other critical elements of the BLA, including a confirmatory study designed to verify and describe the predicted clinical benefit.\nREGENXBIO completed its database lock for the pivotal program and expects to initiate submission of a rolling BLA in the third quarter of 2024. REGENXBIO expects an FDA inspection of its Manufacturing Innovation Center in the first half of 2025. Commercial bulk drug and clinical trial material, both manufactured using REGENXBIO's proprietary NAVXpress™ platform process, were confirmed to be comparable.\nA confirmatory study of RGX-121 is expected to initiate enrollment in the second half of 2025, prior to potential FDA approval. Based on an expected priority review, potential approval of the planned BLA could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025.\n\"This positive engagement with the FDA marks an important milestone for REGENXBIO and RGX-121 on the path towards potential approval of the first gene therapy for Hunter syndrome,\" said Curran Simpson, Chief Operating Officer of REGENXBIO and President and CEO-elect. \"Alignment with the FDA on important elements of this BLA, like our proprietary NAVXpress™ platform process and inspection of our commercial-ready manufacturing facility, provide a solid foundation ...