FDA accepts Vertex' exa-cel BLA for review

9 June 2023

The US Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) that has been submitted by US biotech Vertex Pharmaceutical (Nasdaq: VRTX) and partner Swiss gene editing specialist CRISPR Therapeutics (Nasdaq: CRSP).

Separating review of the two indications, the FDA has granted priority review for SCD and standard review for TDT and assigned Prescription Drug User Fee Act (PDUFA) target action dates of December 8, 2023, and March 30, 2024, respectively. Updated data from the pivotal trials supporting the regulatory submissions will be presented at the Annual European Hematology Association Congress on June 11, 2023.

Reacting to the announcement, Vertex shares edged up 2.4% to $332.58 by close of trading yesterday, while CRISPR rose 6.2% to $66.49 in pre-market activity today.

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