Vertex Pharmaceuticals Incorporated announced that the European Commission has granted conditional marketing authorization to CASGEVY? (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

CASGEVY is the only genetic therapy approved for SCD and TDT patients in the European Union (EU) and with this approval, there are now more than 8,000 patients potentially eligible for treatment. Vertex is already working closely with national health authorities to secure access for eligible patients as quickly as possible. Through this work, Vertex has secured early access for eligible TDT patients in France ahead of the national reimbursement process.

Vertex continues to engage with hospitals experienced in stem cell transplantation to establish a network of independently operated authorized treatment centers (ATCs) for the administration of CASGEVY. There are currently three activated ATCs in the EU and Vertex plans to activate a total of approximately 25 centers across Europe.