European Commission Approves CASGEVY™ for Sickle Cell Disease and Beta Thalassemia

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has announced that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy. CASGEVY is now approved for treating patients aged 12 and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), when hematopoietic stem cell (HSC) transplantation is suitable and a human leukocyte antigen matched related HSC donor is unavailable.

This approval marks CASGEVY as the sole genetic therapy endorsed for SCD and TDT patients in the European Union (EU), potentially benefiting over 8,000 eligible patients.

Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex, emphasized the significance of this approval, indicating that CASGEVY is now authorized for these conditions across multiple regions, offering hope to numerous patients. Kewalramani expressed the company’s commitment to translating these approvals into tangible patient benefits and ensuring global access and reimbursement.

Franco Locatelli, M.D., Ph.D., Principal investigator in the CLIMB-111 and CLIMB-121 studies, highlighted the burdensome nature of SCD and TDT, stressing the importance of providing eligible patients with a therapeutic option like CASGEVY, which offers the potential for a functional cure.

Vertex is actively collaborating with national health authorities to expedite access for eligible patients. The company has already secured early access for eligible TDT patients in France and is establishing a network of authorized treatment centers (ATCs) for CASGEVY administration. Currently, three ATCs are operational in the EU, with plans to activate approximately 25 centers across Europe.

Sickle Cell Disease (SCD) is a debilitating genetic condition characterized by severe pain, organ damage, and reduced life expectancy. Similarly, Transfusion-Dependent Beta Thalassemia (TDT) necessitates lifelong treatment, including frequent blood transfusions, impacting patients’ quality of life and productivity.

The approval of CASGEVY represents a significant milestone in offering potentially transformative treatment options for patients with these challenging conditions, aiming to improve outcomes and enhance overall well-being.

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