Health Canada Oks Vertex's CASGEVY, First CRISPR/Cas9 Gene-Edited Therapy For Sickle Cell Disease

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) said that Health Canada has granted Marketing Authorization for CASGEVY (exagamglogene autotemcel), an autologous genome edited hematopoietic stem cell-based therapy, for the treatment of patients 12 years of age and older with sickle cell disease with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).

The approval is based on the positive interim results from two global and ongoing clinical trials, CLIMB-121 in sickle cell disease and CLIMB-111 in transfusion-dependent beta thalassemia. The primary endpoint of being free from severe VOCs for at least 12 consecutive months or transfusion independence for at least 12 consecutive months was met in both trials. The safety profile is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.

Earlier today, Pfizer Inc. (PFE) announced the voluntary withdrawal of all lots of OXBRYTA (voxelotor) for treating sickle cell disease in all markets where it is approved. It is also discontinuing all active voxelotor clinical trials and expanded access programs worldwide.

For More Such Health News, visit rttnews.com.