Tue. 18 Jun 2024, 7:11am ET
Benzinga
Biotech, News, Health Care, FDA, General
- 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