Casgevy
A single-dose intravenous gene-edited cellular therapy for sickle cell disease and transfusion-dependent beta thalassemia.
Where Exagamglogene Autotemcel is approved
All regulators →Of the three regulators tracked here, the first to approve Exagamglogene Autotemcel was the EU, on 9 Feb 2024. It is approved in 2 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Exagamglogene Autotemcel patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Exagamglogene Autotemcel, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- Casgevy for treatment of beta-thalassaemia intermedia and major: until 12 Feb 2034. Union Register
- Casgevy for treatment of sickle cell disease: until 12 Feb 2034. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 5 Oct 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
How Exagamglogene Autotemcel works
All drug targets →- B-cell lymphoma/leukemia 11A gene editing negative modulator Gene editing negative modulatorTarget: BCL11A BCL11 transcription factor AChEMBL only
- AnemiaEarly phase 1
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL5095474, CC BY-SA 3.0. The Guide to Pharmacology has no record of this medicine, so its targets rest on ChEMBL alone. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Exagamglogene Autotemcel
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 2 technology appraisals of Exagamglogene Autotemcel. 2 are current.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Exagamglogene autotemcel for treating severe sickle cell disease in people 12 years and over NICE TA1044 | Optimised (Innovative Medicines Fund) | 26 Feb 2025 | Current |
| Exagamglogene autotemcel for treating transfusion-dependent beta-thalassaemia in people 12 years and over NICE TA1003 | Recommended (Innovative Medicines Fund) | 11 Sep 2024 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Exagamglogene Autotemcel
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- EUEuropean Union: CasgevyEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
What Casgevy is used for
1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 2 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 2 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). ( 1 ) transfusion-dependent β-thalassemia (TDT). ( 1 )
How it works
12.1 Mechanism of Action 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. In patients with severe sickle cell disease, HbF expression reduces intracellular hemoglobin S (HbS) concentration, preventing the red blood cells from sickling and addressing the underlying cause of disease, thereby eliminating VOCs. In patients with transfusion-dependent β-thalassemia, γ-globin production improves the α-globin to non-α-globin imbalance thereby reducing ineffective erythropoiesis an…
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How this page is built
The facts on this page are pulled directly from official U.S. FDA datasets — they are not written from memory. Each field below names the dataset it came from, so you can check it yourself.
- FDA Drug Label (openFDA) ↗ Indications, mechanism of action and pharmacologic class
- Drugs@FDA ↗ First FDA approval date and the company holding the application
- FDA Drug Shortage Database ↗ Current US supply / shortage status
Plain-English summaries and drug-class explainers are written and reviewed by Sreepriya Prasannan (MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany). Data is retrieved automatically from the sources above and cross-checked with AI-assisted verification (Anthropic's Claude) — brand and generic names are matched against the exact FDA product record so that a combination product or a different formulation cannot be mistaken for the drug on this page. An editor reviews the result before publication. We describe this in full in our editorial standards and corrections policy. The FDA data on this page was last retrieved on 6 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.