Exagamglogene Autotemcel

After CASGEVY infusion, the edited CD34 + cells engraft in the bone marrow and differentiate to erythroid lineage cells with reduced BCL11A expression. Reduced BCL11A expression results in an increase in γ-globin expression and HbF protein production in erythroid cells.

• 1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises transfusion-dependent β - thalassemia (TDT) CASGEVY is an autologous genome edited hematopoietic stem cell-based gene therapy indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs).

• None. None.contraindicated

For autologous use only. For intravenous use only. Patients are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34 + cells for CASGEVY manufacturing. ( 2.2 ) Dosing of CASGEVY is based on body weight. The minimum recommended dose is 3 × 10 6 CD34 + cells/kg. ( 2.1 , 2.3 ) Full myeloablative conditioning must be administered between 48 hours and 7 days before infusion of CASGEVY. ( 2.2 ) Prophylaxis for seizures should be considered prior to initiating myeloablative conditioning. ( 2.2 ) 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. ( 2.2 ) Do not sample, alter, or irradiate CASGEVY. ( 2.2 ) Do not use an in-line blood filter when infusing CASGEVY. ( 2.3 ) Administer each vial of CASGEVY via intravenous infusion within 20 minutes of thaw. ( 2.3 ) 2.1 Dose For autologous use only. For one-time, single dose intravenous use only. The minimum recommended dose of CASGEVY is 3 × 10 6 CD34 + cells/kg. CASGEVY is provided as a single dose for infusion containing a suspension of CD34 + cells in one or more vials. See the Lot Information Sheet provided with the product shipment for additional information pertaining to the number of vials required to achieve the patient-specific dose. Administer all vials. 2.2 Preparation Before CASGEVY Infusion Confirm that hematopoietic stem cell (HSC) transplantation is appropriate for the patient before mobilization, apheresis and myeloablative conditioning are initiated.

Neutrophil Engraftment Failure : Monitor absolute neutrophil counts (ANC) after CASGEVY infusion. Administer rescue cells in the event of neutrophil engraftment failure. ( 5.1 ) Delayed Platelet Engraftment: Monitor platelet counts until platelet engraftment and recovery are achieved. Patients should be monitored for bleeding. ( 5.2 ) Hypersensitivity Reactions : Monitor for hypersensitivity reactions during and after infusion. ( 5.3 ) Off-Target Genome Editing Risk: The risk of unintended, off-target editing in CD34 + cells due to genetic variants cannot be ruled out. ( 5.4 ) 5.1 Neutrophil Engraftment Failure There is potential risk of neutrophil engraftment failure after treatment with CASGEVY. In the clinical trials, all treated patients achieved neutrophil engraftment and no patients received rescue CD34 + cells. Monitor absolute neutrophil counts (ANC) and manage infections according to standard guidelines and medical judgement. In the event of neutrophil engraftment failure, patients should be infused with rescue CD34 + cells [see Adverse Reactions (6.1) ] . 5.2 Delayed Platelet Engraftment Delayed platelet engraftment has been observed with CASGEVY treatment. There is an increased risk of bleeding until platelet engraftment is achieved [see Adverse Reactions (6.1) ] . In the clinical trials, there was no association observed between incidence of bleeding events and time to platelet engraftment. Monitor patients for bleeding according to standard guidelines and medical judgement.

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. ( 6 ) The most common Grade 3 or 4 laboratory abnormalities (≥ 50%) were neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Vertex Pharmaceuticals Incorporated at 1-877-634-8789 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The most common Grade 3 or 4 non-laboratory adverse reactions (occurring in ≥ 25%) were mucositis and febrile neutropenia in patients with SCD and patients with TDT, and decreased appetite in patients with SCD. All (100%) of the patients with TDT and SCD experienced Grade 3 or 4 neutropenia and thrombocytopenia. Other common Grade 3 or 4 laboratory abnormalities (≥ 50%) include leukopenia, anemia and lymphopenia. Sickle Cell Disease The safety of CASGEVY in patients with SCD was evaluated in an open-label, single-arm trial (Trial 1) and a long-term follow-up trial (Trial 3), in which 44 adolescent and adult patients with SCD were treated with CASGEVY after undergoing myeloablative conditioning with busulfan.

Consider the risks of mobilization and myeloablative conditioning agents in patients with reproductive potential and patients that are pregnant or breastfeeding. 8.1 Pregnancy Risk Summary There are no clinical data from the use of exagamglogene autotemcel in pregnant women. No animal reproductive and developmental toxicity studies have been conducted with exagamglogene autotemcel to assess whether it can cause fetal harm when administered to a pregnant woman. CASGEVY must not be administered during pregnancy because of the risks associated with myeloablative conditioning. Pregnancy after CASGEVY infusion should be discussed with the treating physician. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. 8.2 Lactation Risk Summary There are no data on the presence of exagamglogene autotemcel in human or animal milk, the effects on the breastfed child, or the effects on milk production. Because of the potential risks associated with myeloablative conditioning, breastfeeding should be discontinued during conditioning. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CASGEVY and any potential adverse effects on the breastfed child from CASGEVY or from the underlying maternal condition.

Metabolism

CASGEVY is an autologous cellular therapy which includes CD34 + cells that have been edited ex vivo . The nature of CASGEVY is such that conventional studies on pharmacokinetics, absorption, distribution, metabolism, and elimination are not applicable.

1. 1Sickle Cell Anaemia With Crisis413%
2. 2Infusion Related Reaction310%
3. 3Abdominal Pain13.3%
4. 4Alopecia13.3%
5. 5Apheresis Related Complication13.3%
6. 6Appendicitis13.3%
7. 7Asthenia13.3%
8. 8Cancer Screening Abnormal13.3%
9. 9Cardiac Arrest13.3%
10. 10Catheter Site Thrombosis13.3%
11. 11Colitis Ulcerative13.3%
12. 12Complication Associated With Device13.3%
13. 13Device Related Thrombosis13.3%
14. 14Diarrhoea13.3%
15. 15Dizziness13.3%

The 9 most recently updated of 9 ClinicalTrials.gov registrations naming Exagamglogene Autotemcel as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.
  Recruiting · Early phase 1 · Interventional · 72 enrolled · Cajal Therapeutics Inc.
  NCT07577817updated 2026-06-12
• An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)
  Recruiting · Phase 1 · Phase 2 · Interventional · 75 enrolled · Complement Therapeutics
  NCT07392255updated 2026-05-04
• A Long-term Follow-up Study in Participants Who Received CTX001
  Enrolling by invitation · Phase 3 · Interventional · 160 enrolled · Vertex Pharmaceuticals Incorporated
  NCT04208529updated 2026-03-25
• Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
  Withdrawn · Phase 3 · Interventional · 0 enrolled · Vertex Pharmaceuticals Incorporated
  NCT05951205updated 2026-03-25
• Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
  Recruiting · Phase 3 · Interventional · 26 enrolled · Vertex Pharmaceuticals Incorporated
  NCT05477563updated 2026-03-23
• Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
  Active not recruiting · Phase 3 · Interventional · 13 enrolled · Vertex Pharmaceuticals Incorporated
  NCT05329649updated 2026-03-09
• Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
  Active not recruiting · Phase 3 · Interventional · 16 enrolled · Vertex Pharmaceuticals Incorporated
  NCT05356195updated 2026-03-03
• A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
  Completed · Phase 2 · Phase 3 · Interventional · 59 enrolled · Vertex Pharmaceuticals Incorporated
  NCT03655678updated 2025-12-17
• A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
  Completed · Phase 2 · Phase 3 · Interventional · 63 enrolled · Vertex Pharmaceuticals Incorporated
  NCT03745287updated 2025-08-11

Frequently asked questions

How does Exagamglogene Autotemcel work?

After CASGEVY infusion, the edited CD34 + cells engraft in the bone marrow and differentiate to erythroid lineage cells with reduced BCL11A expression. Reduced BCL11A expression results in an increase in γ-globin expression and HbF protein production in erythroid cells.

What is Exagamglogene Autotemcel used for?

According to FDA labeling, Exagamglogene Autotemcel carries indications including: 1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises transfusion-dependent β - thalassemia (TDT) CASGEVY is an autologous genome edited hematopoietic stem cell-based gene therapy indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs).. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Exagamglogene Autotemcel?

Exagamglogene Autotemcel is classified as Other hematological agents, Gene Editing or Modification, Cellular Structure Alteration.

What are the brand names for Exagamglogene Autotemcel?

Exagamglogene Autotemcel is marketed under brand names including Casgevy.

What are the contraindications for Exagamglogene Autotemcel?

Exagamglogene Autotemcel labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.