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Drug

Exagamglogene autotemcel

Approved medicine
ClassB-cell lymphoma/leukemia 11A gene editing negative modulatorResearchOngoing clinical research

Also known as Casgevy, Exa-cel, Autologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA).

RxNorm2671667ChEMBLCHEMBL5095474ATCB06AX05UNIIS53L777GM8
BCL11 transcription factor A
Primary target
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Exagamglogene autotemcel
Aliases & brands
CasgevyExa-celAutologous CD34+ cells isolated from mobilised peripheral blood by positive selection, modified by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a guide RNA (gRNA)
RxNorm CUI
2671667
ChEMBL ID
CHEMBL5095474
ATC codes
B06AX05
UNII
S53L777GM8
Primary mechanism
B-cell lymphoma/leukemia 11A gene editing negative modulator
Regulatory jurisdictions
ema

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

BCL11 transcription factor A
BCL11A · Q9H165
GENE EDITING NEGATIVE MODULATORB-cell lymphoma/leukemia 11A gene editing negative modulator

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2024-02-09
Latest approval
2024-02-09
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2024-02-09Approval
Approval: Casgevy (EMA)
Indication: β thalassemia Casgevy is indicated for the treatment of transfusion dependent β thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC)… Show full indication

β thalassemia Casgevy is indicated for the treatment of transfusion dependent β thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available. Sickle cell disease Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

1 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
1
registered trials across all phases
1
Late-stage (III+)
1
Discontinued
PHASE DISTRIBUTIONn = 1
Phase 31

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects With Severe Sickle Cell Disease, βS/βC Genotype

WithdrawnPH IIIVertex Pharmaceuticals Incorporated2033
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.