Casgevy (exagamglogene autotemcel) — Medica

Initial criteria

• Patient is age ≥ 12 years
• Patient has not received a gene therapy for sickle cell disease in the past [verification in claims history required]; if claims history unavailable, prescribing physician confirms no prior Casgevy or Lyfgenia use
• According to the prescribing physician, a hematopoietic stem cell transplantation is appropriate for the patient
• Patient meets ONE of the following: (i) does not have a Human Leukocyte Antigen (HLA)-matched donor; OR (ii) has an HLA-matched donor but the individual is not able or not willing to donate
• Genetic testing [documentation required] demonstrates ONE of: (i) βS/βS genotype; OR (ii) βS/β0 genotype; OR (iii) βS/β+ genotype (other genotypes reviewed case‑by‑case by Medical Director)
• Patient has tried at least ONE pharmacologic treatment for sickle cell disease [documentation required] (examples: hydroxyurea, L‑glutamine, Adakveo, Oxbryta)
• While receiving appropriate standard treatment, patient had ≥4 severe vaso‑occlusive crises/events in the previous 2 years [documentation required]
• Patient does NOT have ANY of the following: (i) clinically significant and active bacterial, viral, fungal, or parasitic infection; (ii) advanced liver disease [documentation required]; (iii) severe cerebral vasculopathy (untreated or high‑risk Moyamoya); (iv) prior or current malignancy, myeloproliferative disorder, or significant immunodeficiency disorder
• Prescribing physician confirms ALL of the following medication discontinuations: (i) disease‑modifying therapies for sickle cell disease stopped ≥2 months before mobilization/conditioning; (ii) iron chelation therapy stopped ≥7 days before myeloablative conditioning
• Prescribing physician confirms ALL of the following procedures: (i) patient will undergo mobilization, apheresis, and myeloablative conditioning; (ii) a hematopoietic stem cell mobilizer (e.g., Mozobil) will be utilized; (iii) Busulfan will be used for myeloablative conditioning; (iv) sickle hemoglobin <30% with total hemoglobin ≤11 g/dL prior to mobilization and until initiation of conditioning
• Screening negative [documentation required] for ALL: human immunodeficiency virus‑1/‑2, hepatitis B virus, hepatitis C virus, and human T‑lymphotrophic virus‑1/‑2 (permitted per HBV note if vaccinated or past exposure but HBV DNA negative)
• Reproductive potential requirement: (i) females: negative serum pregnancy test prior to each mobilization cycle and again before conditioning AND effective contraception from start of mobilization through 6 months post‑dose; OR (ii) males: effective contraception from start of mobilization through 6 months post‑dose
• Medication is prescribed by a hematologist or a stem cell transplant physician
• Current patient body weight obtained within 30 days [documentation required]
• If all above criteria met, approve one intravenous dose (one‑time per lifetime) containing ≥3 × 10⁶ CD34+ cells/kg [verification required]

Approval duration

1 year (to allow preparation and administration of single lifetime dose)