Approvals

Vertex Pharmaceuticals has announced that the European Commission (EC) has granted conditional marketing authorisation to Casgevy (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy.

Casgevy is approved for the treatment of patients over the age of 12 with severe sickle cell disease (SCD) characterised by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), in patients for whom haematopoietic stem cell (HSC) transplantation is appropriate and when a human leukocyte antigen matched related HSC donor is not available.

The drug is now the only genetic therapy available for SCD and TDT patients in the EU following this approval.

Reshma Kewalramani MD, chief executive officer and president of Vertex, commented: “With this approval, Casgevy is now approved for SCD and TDT in multiple geographies making tens of thousands of patients eligible for this potentially transformative therapy. Now our goal shifts to translating these approvals into real-world patient benefit and ensuring access and reimbursement across the globe.”

Franco Locatelli MD PhD, principal investigator in the CLIMB-111 and CLIMB-121 studies, professor of paediatrics at the Catholic University of the Sacred Heart, Rome, Italy, and director of the Department of Pediatric Hematology and Oncology at the Bambino Gesù Children’s Hospital, Italy, added: “SCD and TDT are debilitating, life-shortening diseases associated with significant burden on patients, families and healthcare systems. 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.”

The company is already working with the relevant health authorities to ensure the drug is available to those who need it as quickly as possible, and according to the company’s press release: ‘Vertex is already working closely with national health authorities to secure access for eligible patients as quickly as possible’.