Exa-cel Gene-Edited Therapy
Vertex Pharmaceutical recently announced that Health Canada has accepted its New Drug Submission and granted Priority Review for Exa-cel, a gene-edited cell therapy, for the treatment of sickle cell disease and beta thalassemia.
Priority Review status grants eligible submissions 180 days in lieu of the conventional 300-day timeline. The announcement, published recently in the Malaysian Review, also included Health Canada’s acceptance of Exa-cel developed for the treatment of patients 12 through age 35 diagnosed with transfusion-dependent beta thalassemia. The submission was based on results of the Phase III studies, CLIMB-III and CLIMB-l2l. The list also includes CLIMB-l3l, the long-term ongoing follow-up. The Phase III studies were recently featured at the annual meeting of ASH.
About Exagamglogene Autotemcel (exa-cel)
Exa-cel, known previously as CTX001, is a gene-edited therapy being assessed to treat patients with sickle cell disease and transfusion dependent beta thalassemia. Exa-cel’s elevation of fetal hemoglobin shows its potential to lessen the number of transfusions required for patients thereby reducing debilitating and painful sickle crises.
About Sickle Cell Disease
Sickle cell disease (SCD) is a life shortening, debilitating, genetic disease that affects red blood cells, which transport oxygen to all tissues and organs in the body. Severe pain and organ damage is caused by the misshaped “sickled” red blood cells. Although stem cell transplant from a matched donor is curative, only a small number of people are able to use this option.
About Transfusion-Dependent Beta Thalassemia (TDT)
Iron chelation therapy and blood transfusions are the most common treatments for people living with transfusion-dependent beta thalassemia. It is a serious disease that can reduce life expectancy. Complications may include enlarged liver, spleen or heart and misshapen bones.
Similar to SCD, a stem cell transplant is curative but again only in a few select cases.
The efficacy and safety of Exa-cel has not as yet been established by the Health Technology Assessment organizations, the CADTH, or INESSS in Quebec. The trials are now closed for enrollment. Patients will be monitored for about two years after exa-cel infusion.
