Casgevy®

Drug - Casgevy® (exagamglogene autotemcel) [Vertex Pharmaceuticals Inc.]

February 2026

Therapeutic Area - Sickle Cell Disease (SCD) and Transfusion-Dependent Beta-Thalassemia (TDT)

Sickle Cell Disease (SCD)

Approval criteria

Patient is at least twelve (12) years of age; AND
Patient has prior use of, or intolerance to hydroxyurea (per health care professional judgement) at any point in the past; AND
Patient is clinically stable and fit for transplantation; AND
Patient has been screened and found negative for hepatitis B virus (HBV), hepatitis C virus (HCV), and human immunodeficiency virus 1 & 2 (HIV-1/HIV-2) in accordance with clinical guidelines prior to collection of cells (leukapheresis); AND
Patient has not received other gene therapies for sickle cell disease [e.g., Lyfgenia® (lovotibeglogene autotemcel)]; AND
Patient is a candidate for autologous HSCT and has not had prior HSCT; AND
Patient has a confirmed diagnosis of sickle cell disease with confirmatory genetic testing; AND
Patient has experienced recurrent VOCs* (defined as more than or equal to two (2) documented VOCs per year in the previous twenty-four (24) months, based on provider attestation); AND
Casgevy is prescribed in consultation with a board-certified hematologist with SCD expertise

*Vaso-Occlusive Crisis (“VOC”): A VOC occurs when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen. This in turn sets in motion an inflammatory response as the body tries to rectify the problem.

Transfusion-Dependent Beta-Thalassemia (TDT)

Approval criteria

Patient is at least twelve (12) years of age; AND
Patient is clinically stable and fit for transplantation; AND
Patient has been screened and found negative for hepatitis B virus (HBV), hepatitis C virus (HCV), and human immunodeficiency virus 1 & 2 (HIV-1/HIV-2) in accordance with clinical guidelines prior to collection of cells (leukapheresis); AND
Patient has not received other gene therapies for TDT [e.g., Zynteglo® (betibeglogene autotemcel)]; AND
Patient is a candidate for autologous HSCT and has not had prior HSCT; AND
- Patient has a confirmed diagnosis of beta-thalassemia (including hemoglobin E beta thalassemia) with confirmatory genetic testing; OR
- Patient has severe microcytic hypochromic anemia, absence of iron deficiency, anisopoikilocytosis with nucleated red blood cells on peripheral blood smear, and hemoglobin analysis that reveals decreased amounts or complete absence of hemoglobin A (HbA) and increased HbA2 with or without increased amounts of hemoglobin F (HbF);
Patient has transfusion-dependent beta-thalassemia disease defined as a history of transfusions of ≥ 100 mL/kg/year or ≥10 units/year of packed red blood cells (pRBCs) in the 2 years preceding therapy; AND
Patient will be transfused prior to achieving a total Hb ≥ 11 g/dL for 60 days prior to myeloablative conditioning; AND
Patient must NOT have any of the following:
- Severely elevated iron in the heart (e.g., patients with cardiac T2* < 10 msec by magnetic resonance imaging [MRI] or left ventricular ejection fraction [LVEF] < 45% by echocardiogram); OR
- Advanced liver disease (e.g., aspartate transaminase [AST] or alanine transaminase [ALT] > 3 times the upper limit of normal [ULN], or direct bilirubin value > 2.5 times the ULN, or if a liver biopsy demonstrated bridging fibrosis or cirrhosis).
Casgevy is prescribed in consultation with a board-certified hematologist with TDT expertise

Duration of approval

Prior authorization approval is effective for 12 months from the approval date. Approval may be extended for another 6 months if patient is unable to receive treatment within 12 months from the approval date.

Quantity limits

1 administration per lifetime
Patient’s most current weight (in kg) and the dose to be administered must be provided at time of request

Billing for Casgevy

Casgevy must be billed as a professional claim

Questions

Provider Call Center: (844) 575-7887