European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today 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.

“With this approval, CASGEVY is now approved for sickle cell disease and transfusion-dependent beta thalassemia in multiple geographies making tens of thousands of patients eligible for this potentially transformative therapy,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “Now our goal shifts to translating these approvals into real-world patient benefit and ensuring access and reimbursement across the globe.”

“Sickle cell disease and transfusion-dependent beta thalassemia are debilitating, life-shortening diseases associated with significant burden on patients, families and health care systems,” said Franco Locatelli, M.D., Ph.D., Principal investigator in the CLIMB-111 and CLIMB-121 studies, Professor of Pediatrics at the Catholic University of the Sacred Heart, Rome, and Director of the Department of Pediatric Hematology and Oncology at the Bambino Gesù Children’s Hospital. “CASGEVY offers the potential of a functional cure, and it will be important to offer this therapeutic option to eligible patients as soon as possible.”

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.