European Commission Approves Conditional MA for Sickle Cell Disease, Transfusion-dependent Beta Thalassemia

The European Commission has approved the Conditional Marketing Authorization (MA) for exagamglogene autotemcel (exa-cel; CASGEVY), according to the therapy’s developer, Vertex Pharmaceuticals.

Exa-cel is now approved for patients aged 12 years and older with either severe sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia eligible for hematopoietic stem cell transplantation without a human leukocyte antigen-matched related donor.

Vertex Pharmaceuticals has secured early access to exa-cel for eligible patients with transfusion-dependent beta thalassemia in France, and there are three active authorized treatment centers out of a planned 25 in the European Union.

“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,” said Franco Locatelli, MD, PhD, the Principal Investigator of the CLIMB-111 and CLIMB-121 studies.

Related: Haydar Frangoul, MD, Details CLIMB-121 Data on Exa-Cel in Sickle Cell Disease