Exagamglogene autotemcel
As of the edition of 20 October 2026, Exagamglogene autotemcel is a gene: marketed, first approved in 2023 (US); acts on BCL11A; US protection ends in 2035.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Marketed; latest approval 2024
- Kind
- Gene
- Brand name
- Casgevy
- First approved
- 8 December 2023 (US)
- Approvals on record
- 3 in 3 regions
- Holder of the most recent approval
- Vertex; 2 holders in all
- Registered trials
- 8, 1 stopped early
- Disease areas
- Blood and lymph; Inherited and neonatal; Nutrition and metabolism; Pregnancy and women's health; Urinary and reproductive
US protection
US protection ends in 2035.
US protection ends in 2035, computed as 12 years from the first US licence because FDA publishes no date for this product. The application that gives this date is held by Vertex. The date is computed, not published.
This is the US only, and it is not the date generics or biosimilars can enter.
The exact date and the listings behind it
In the product2 listings count towards this date. The exact date and each listing are in the product.
Companies that need a new molecule because of this drug
1 company on route B because of this drug.
Companies on route B for this drug
In the product1 company is on route B because of Exagamglogene autotemcel.
Target
| Gene | Protein | UniProt |
|---|---|---|
| BCL11A | BCL11 transcription factor A | Q9H165 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase 1 | 1 | 0 | 1 | 0 |
| Phase 3 | 7 | 2 | 4 | 1 |
| All trials | 8 |
Who has held its approvals
| Company | Role | Years |
|---|---|---|
| Vertex | Originator, Applicant and Lead sponsor of trials | 2018 to now |