Exagamglogene Autotemcel (Casgevy) – Guidelines+ Monograph

Since 2023, the autologous genome edited hematopoietic stem cell-based  gene therapy (Casgevy) exagamglogene autotemcel has been indicated for sickle cell disease with recurrent vaso-occlusiev crises (VOCs). A year later, in 2024, Casgevy received an expanded indication to include its use as treatment of transfusion-dependent β-thalassemia. 

Read on to learn more about Casgevy, including its warnings and precautions, dosage and administration information, and more. 

Medication Overview:

• Brand Name: Casgevy
• Generic Name: exagamglogene autotemcel
• Treatment for: Sickle cell disease and transfusion-dependent β-thalassemia
• Company: Vertex Pharmaceuticals
• Initial FDA Approval: December 2023

Warnings and Precautions:

• Neutrophil Engraftment Failure: Monitor absolute neutrophil counts (ANC) after Casgevy infusion. Administer rescue cells in the event of neutrophil engraftment failure.
• Delayed Platelet Engraftment: Monitor platelet counts until platelet engraftment and recovery are achieved. Patients should be monitored for bleeding.
• Hypersensitivity Reactions: Monitor for hypersensitivity reactions during and after infusion.
• Off-Target Genome Editing Risk: The risk of unintended, off-target editing in CD34+cells due to genetic variants cannot be ruled out.

Dosage and Administration:

• Patients are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for Casgevy manufacturing.
• Dosing of Casgevy is based on body weight. The minimum recommended dose is 3 × 106 CD34+ cells/kg.
• Full myeloablative conditioning must be administered between 48 hours and 7 days before infusion of Casgevy.
• Prophylaxis for seizures should be considered prior to initiating myeloablative conditioning.
• Verify that the patient's identity matches the unique patient identification information on the product labels and Lot Information Sheet prior to thaw and infusion.
• Do not sample, alter, or irradiate Casgevy.
• Do not use an in-line blood filter when infusing Casgevy.
• Administer each vial of Casgevy via intravenous infusion within 20 minutes of thaw.

Contraindications:

None. 

Drug Interactions:

Granulocyte-Colony Stimulating Factor: Granulocyte-Colony Stimulating Factor (G-CSF) must not be used for CD34+ HSC mobilization of patients with SCD.

Hydroxyurea: Discontinue hydroxyurea at least 8 weeks prior to start of mobilization and conditioning.

Voxelotor and Crizanlizumab: Discontinue the use of voxelotor and crizanlizumab at least 8 weeks prior to start of mobilization and conditioning.

Iron Chelators: Discontinue iron chelators at least 7 days prior to initiation of myeloablative conditioning. Avoid the use of non-myelosuppressive iron chelators for at least 3 months and use of myelosuppressive iron chelators for at least 6 months after Casgevy infusion. 

Adverse Reactions:

The most common Grade 3 or 4 non-laboratory adverse reactions (incidence ≥ 25%) were mucositis and febrile neutropenia in patients with SCD and TDT, and decreased appetite in patients with SCD.

The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.

Related Guidelines

• Gene Therapy for Sickle Cell Disease
  • American Society for Transplantation and Cellular Therapy (ASTCT), International Society for Cell and Gene Therapy (ISCT)
  • March 2026

• Management of Transfusion-Dependent β-Thalassaemia
  • Thalassaemia International Federation
  • January 2025

Please note: This article is current as of June 12, 2026. Consult our clinical guidelines library or drug information tool to ensure you always have the most up-to-date information.

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