Lyfgenia (lovotibeglogene autotemcel) — Medica

Initial criteria

• Patient has sickle cell disease genotype for which Lyfgenia is indicated (other genotypes reviewed case-by-case by Medical Director)
• age ≥ 12 years
• Patient has tried at least ONE pharmacologic treatment for sickle cell disease (e.g., hydroxyurea, L-glutamine, Adakveo [crizanlizumab-tmca], or Oxbryta [voxelotor])
• While receiving appropriate standard treatment for sickle cell disease, patient had at least four severe vaso-occlusive crises or events in the previous 2 years
• Patient does not have: more than two α-globin gene deletions; clinically significant and active infection; advanced liver disease; severe cerebral vasculopathy (e.g., untreated Moyamoya disease or presence of Moyamoya disease at risk of bleeding per physician); prior/current malignancy, myeloproliferative disorder, or significant immunodeficiency disorder
• Patient will have been discontinued from: disease-modifying therapies for sickle cell disease for ≥ 2 months; erythropoietin for ≥ 2 months; iron chelation therapy for ≥ 7 days; and antiretroviral prophylaxis for ≥ 1 month prior to mobilization
• Patient will undergo mobilization, apheresis, and myeloablative conditioning; a hematopoietic stem cell mobilizer (e.g., Mozobil [plerixafor]) will be utilized; busulfan will be used for myeloablative conditioning; sickle hemoglobin level < 30% of total hemoglobin and total hemoglobin ≤ 11 g/dL prior to mobilization and until initiation of conditioning
• Screening is negative for HIV-1/2, hepatitis B virus, hepatitis C virus, and human T-lymphotrophic virus-1/2
• If of reproductive potential: females have negative pregnancy tests before mobilization cycles and prior to myeloablative conditioning and use effective contraception from start of mobilization through at least 6 months after Lyfgenia; males use effective contraception from start of mobilization through at least 6 months after Lyfgenia
• Medication prescribed by a hematologist or stem cell transplant physician
• Current body weight obtained within 30 days
• Patient has not received prior hematopoietic stem cell transplantation
• Patient has not received prior gene therapy (e.g., Casgevy [exagamglogene autotemcel])

Approval duration

one-time lifetime dose