Vertex Pharmaceuticals Inc. (VRTX) has announced that Health Canada has granted Marketing Authorization for CASGEVY (exagamglogene autotemcel), an autologous genome-edited hematopoietic stem cell-based therapy. This therapy is intended for patients aged 12 and older who suffer from sickle cell disease with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta-thalassemia (TDT).
The authorization is supported by interim positive results from two ongoing global clinical trials: CLIMB-121 for sickle cell disease and CLIMB-111 for transfusion-dependent beta-thalassemia. In both trials, the primary endpoint was achieved, with patients being free from severe VOCs for at least 12 consecutive months or maintaining transfusion independence for at least 12 consecutive months. The safety profile reported aligns with the expectations for myeloablative conditioning using busulfan and hematopoietic stem cell transplantation.
Separately, Pfizer Inc. (PFE) has announced the voluntary withdrawal of all lots of OXBRYTA (voxelotor) for the treatment of sickle cell disease across all approved markets. Additionally, Pfizer is terminating all ongoing voxelotor clinical trials and expanded access programs worldwide.
