CASGEVY (exagamglogene autotemcel) (Exagamglogene Autotemcel INJECTION, SUSPENSION) 13000000 1/mL

CASGEVY (exagamglogene autotemcel) is an FDA-approved gene therapy developed by Vertex Pharmaceuticals for treating sickle cell disease and transfusion-dependent beta-thalassemia. It represents a breakthrough in cell and gene therapy, utilizing CRISPR gene-editing technology to modify patients' own hematopoietic stem cells. This orphan drug offers a potentially curative approach for patients with severe hemoglobinopathies.

Strength

13000000 1/mL

Pack Size

9 VIAL in 1 CARTON (51167-290-09) / 20 mL in 1 VIAL (51167-290-01)

Manufacturer / TM Owner

Vertex Pharmaceuticals

Select Quantity

Pack/s

Request a Quote

Please fill out the form below and our team will get back to you shortly with pricing and further information.

First Name

Last Name

Phone

Delivery Country

Delivery PostCode/Zipcode

Select Your Profile

I have read and agree to the terms & conditions, including the privacy policy, return policy, and usage terms as applicable to this request.

Indications & Clinical Uses

CASGEVY is indicated for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) in patients with severe disease manifestations. The drug targets adult and pediatric patients who experience recurrent vaso-occlusive crises, chronic hemolysis, and transfusion dependency. CASGEVY works through ex vivo CRISPR/Cas9 gene editing of autologous CD34+ hematopoietic stem cells, which are modified to produce high levels of fetal hemoglobin (HbF). These edited cells are then reinfused into the patient following myeloablative conditioning, allowing engraftment and sustained production of HbF-expressing red blood cells. This mechanism effectively reduces sickling and hemolysis, potentially eliminating the need for chronic transfusions and reducing vaso-occlusive crisis frequency in eligible patients.

Side Effects

Common adverse events associated with CASGEVY include those related to the conditioning regimen and stem cell transplantation process, including cytopenias, infections, and infusion-related reactions. Serious adverse events may include severe infections, graft failure, secondary malignancies, and immune-mediated complications. Patients require intensive monitoring including complete blood counts, infectious disease surveillance, and long-term follow-up for potential genotoxicity. Regular hematologic assessments and clinical evaluation for vaso-occlusive crises and transfusion requirements are essential during the recovery and engraftment phases.

Precautions & Warnings

CASGEVY is contraindicated in patients with active infections or significant comorbidities that preclude myeloablative conditioning. Pregnancy is a contraindication due to the myeloablative conditioning requirement. Pediatric patients require careful evaluation of disease severity and conditioning tolerance. Patients with hepatic or renal impairment may require dose adjustments or enhanced monitoring. The drug carries black box warnings regarding potential genotoxicity and secondary malignancy risk from CRISPR gene editing. Informed consent regarding long-term safety data and unknown risks is mandatory. Patients must be counseled on fertility implications and contraception requirements.

Avoid Interactions

CASGEVY has no direct drug-drug interactions as it is administered as a cellular therapy product. However, concurrent medications affecting bone marrow function, immunosuppression, or hematopoiesis should be reviewed and potentially discontinued prior to conditioning. Patients receiving CASGEVY should avoid live vaccines during the conditioning and early post-transplantation period. Medications that impair renal or hepatic function may affect conditioning drug metabolism and should be used cautiously. Coordination with oncology and hematology specialists is essential for managing potential interactions with supportive care medications.

Frequently Asked Questions

CASGEVY is used to treat sickle cell disease and transfusion-dependent beta-thalassemia in patients with severe disease. It is a gene therapy that modifies the patient's own stem cells using CRISPR technology to produce fetal hemoglobin, reducing sickling and hemolysis. This can potentially eliminate the need for chronic transfusions and reduce vaso-occlusive crises.

CASGEVY is administered intravenously as a one-time infusion following myeloablative conditioning chemotherapy. The patient's CD34+ hematopoietic stem cells are first collected, edited ex vivo using CRISPR technology, and then reinfused intravenously. The entire process requires hospitalization and intensive monitoring during the conditioning and engraftment phases.

Common side effects include cytopenias (low blood cell counts), infections, and infusion-related reactions associated with the conditioning regimen and stem cell transplantation. Patients may experience fatigue, nausea, and other effects typical of myeloablative conditioning. Serious complications such as severe infections and graft failure require close medical monitoring.

CASGEVY is manufactured by Vertex Pharmaceuticals, a leading biopharmaceutical company specializing in innovative therapies for serious diseases.

Yes, CASGEVY has been designated as an orphan drug by the FDA for both sickle cell disease and transfusion-dependent beta-thalassemia. This designation recognizes that these are rare diseases affecting a limited patient population and provides regulatory incentives for development and commercialization of therapies addressing these serious conditions.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated: 17 Jun 2026

Disclaimer

At GNH India, we aim to provide information that is expert-reviewed, accurate, and reliable. However, all content on this platform is intended for general informational purposes only and does not constitute professional medical advice, diagnosis, or treatment. While efforts are made to maintain accuracy and currency, the information may contain errors, omissions, or may not reflect the latest developments. Always consult a qualified healthcare professional regarding any medical concerns.

All product names, brand names, trademarks and registered trademarks displayed on this website are the property of their respective owners. This website and its operator are not affiliated with, endorsed by, or authorized by any such trade mark owners unless expressly stated. Any reference to product names or trademarks is made solely for descriptive and informational purposes. No commercial relationship, sponsorship, approval, or association with any trademark owner should be inferred. Information provided herein does not constitute promotion, advertising, or an offer for sale of any medicinal product and is intended solely for general informational purposes.

All trademarks and brand names displayed on this website are used strictly in accordance with the provisions of the Trade Marks Act, 1999, specifically Sections 30 and 30(1) governing fair use. Trademark ownership has been accurately attributed under the "Manufacturer/ TM Owner" section in compliance with statutory requirements.