FDA accepts exa-cel BLAs; pivotal trials meet primary endpoints in SCD and TDT

CRISPR Therapeutics AG

FDA granted Priority Review for SCD (PDUFA Dec 8, 2023) and Standard Review for TDT (PDUFA Mar 30, 2024).
In TDT, 24/27 (88.9%) achieved transfusion-independence ≥12 months; mean duration 20.5 months.
In SCD, 16/17 (94.1%) achieved freedom from vaso-occlusive crises ≥12 months; mean duration 18.7 months.
Safety profile consistent with busulfan conditioning; no deaths or discontinuations related to exa-cel.
Data from 83 patients (48 TDT, 35 SCD) with follow-up up to 43.7 months presented at EHA 2023.

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