Exagamglogene autotemcel (Casgevy)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Casgevy be reimbursed by public drug plans for the treatment of transfusion-dependent beta-thalassemia (TDT) if certain conditions are met.
• Casgevy should only be covered to treat patients 12 years of age and older with a diagnosis of TDT (defined as documented homozygous or compound heterozygous beta-thalassemia, and having received transfusions of packed red blood cells [RBCs] of at least 100mL/kg/year, or 10 units/year, during the previous 2 years), who meet specific Karnofsky (patients 16 years or older) or Lansky (patients under 16 years of age) performance status thresholds, who are eligible for autologous stem cell transplant, do not have an available and willing 10/10 HLA-matched related donor, have not previously received allogenic hematopoietic stem cell transplant (allo-HSCT) or prior gene editing therapy or gene editing products, and who do not have any of the following: associated alpha thalassemia and 1 or more alpha deletions or alpha multiplications, prior or current history of malignancy, or sickle cell beta-thalassemia variant.
• Casgevy should only be reimbursed if prescribed by a hematologist with expertise in TDT, if it is not a re-treatment (Casgevy is a 1-time treatment), and the cost of Casgevy is reduced.