regulatory
confidence high
sentiment positive
materiality 0.80
FDA grants rolling review for CRISPR/Vertex exa-cel; submissions planned Q4 2022 / Q1 2023
CRISPR Therapeutics AG
- FDA granted rolling review for exa-cel BLA for sickle cell disease and transfusion-dependent beta thalassemia.
- BLA submission starts November 2022; completion expected by end of Q1 2023.
- EMA and MHRA submissions on track for submission by end of 2022.
- Exa-cel holds FDA RMAT, Fast Track, Orphan Drug designations; EMA PRIME and Orphan Drug.