Exa-Cel Approved in Saudi Arabia for Sickle Cell Disease, Beta Thalassemia

The Saudi Food and Drug Authority (SFDA) granted Marketing Authorization to exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), according to Vertex Pharmaceuticals, the manufacturer of the drug.

Exa-cel, developed under the trade name, CASGEVY, is a gene-editing therapy that utilizes the CRISPR/Cas9 technology to modify the erythroid specifier enchancer region of the BCL11A gene. The therapy allows patients to produce fetal hemoglobin, a form of oxygen carrying hemoglobin, that leads to reduced or eliminated vaso-occlusive crises, according to the company.

“This approval adds to the list of firsts for CASGEVY. It represents the first medicine ever to receive SFDA Breakthrough Designation and be approved through this pathway, and Vertex’s first ever regulatory approval in the Kingdom of Saudi Arabia,” said Reshma Kewalramani, MD, Chief Executive Officer and President of Vertex.

Notably, the Kingdom of Saudi Arabia has some of the highest rates of SCD and TDT in the world, according to Vertex’s release. The Ministry of National Guard Health Affairs is the first Authorized Treatment Center in Saudi Arabia, and Vertex is working to increase qualified centers.

“Most importantly, with this approval, people living with sickle cell disease or transfusion-dependent beta thalassemia have the possibility of a one-time transformative therapy for their disease,” Dr. Kewalramani noted.