European Commission grants conditional marketing authorization for Casgevy

CRISPR Therapeutics/Casgevy (exa-cel)

On 13 February 2024 the European Commission granted Casgevy conditional marketing authorisation for sickle cell disease and transfusion-dependent beta thalassemia, opening the EU market to the first CRISPR-edited cell therapy.

• Casgevy edits patient blood stem cells to restore fetal hemoglobin production, providing long-term benefit from one-time treatment.
• Approved for patients ≥12 years with SCD or TDT; over 8,000 patients in Europe potentially eligible.
• Conditional authorization is based on clinical evidence and post-approval monitoring; full authorization requires additional data.
• EU approval followed UK (November 2023) and Bahrain (December 2023); US FDA approved in December 2023.