Casgevy (exagamglogene autotemcel) — Medica

Initial criteria

• age ≥ 12 years
• Has not received a gene therapy for beta-thalassemia in the past (no prior Casgevy or Zynteglo)
• According to the prescribing physician, a hematopoietic stem cell transplantation is appropriate
• Patient does NOT have an HLA-matched donor OR has an HLA-matched donor who is not able or willing to donate
• Has a genotype confirmed by genetic testing: non-β0/β0 or β0/β0
• Is transfusion-dependent, defined by: receipt of transfusions ≥ 100 mL/kg/year in previous 2 years OR ≥ 10 units/year in previous 2 years
• Evaluated for severe iron overload AND does not have evidence of severe iron overload
• No active bacterial, viral, fungal, or parasitic infection
• No prior or current malignancy, myeloproliferative disorder, or significant immunodeficiency disorder, AND no advanced liver disease
• Iron chelation therapy discontinued ≥ 7 days prior to myeloablative conditioning
• Will undergo mobilization, apheresis, and myeloablative conditioning using a granulocyte-colony stimulating factor, a hematopoietic stem cell mobilizer (e.g., Mozobil), and busulfan
• Total hemoglobin ≥ 11 g/dL prior to mobilization and prior to myeloablative conditioning
• Screening negative for HIV-1/2, hepatitis B, hepatitis C, and human T-lymphotropic virus-1/2
• For patients of reproductive potential: females have negative serum pregnancy test before mobilization and before conditioning, and both males and females use effective contraception from mobilization through 6 months after Casgevy administration
• Prescribed by a hematologist or stem cell transplant physician
• Current body weight obtained within 30 days

Approval duration

one-time (per lifetime) single dose