Last reviewed · How we verify
Leukeran (CHLORAMBUCIL)
Leukeran works by attaching an alkyl group to the DNA of cancer cells, interfering with their ability to replicate and ultimately leading to cell death.
Leukeran (CHLORAMBUCIL) is a small molecule alkylating drug originally developed by ASPEN GLOBAL INC and currently owned by Waylis Therap. It targets Cathepsin D and was FDA approved in 1957 for the treatment of Chronic lymphoid leukemia, Follicular non-Hodgkin's lymphoma, and Hodgkin's disease. Leukeran is an off-patent medication with no active Orange Book patents, meaning it is available as a generic. The drug has a half-life of 1.1 hours and bioavailability of 82%. Key safety considerations include its potential for myelosuppression and gastrointestinal toxicity.
At a glance
| Generic name | CHLORAMBUCIL |
|---|---|
| Sponsor | Waylis |
| Drug class | Alkylating Drug [EPC] |
| Target | Cathepsin D |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 1957 |
Mechanism of action
Mechanism of Action. Chlorambucil, an aromatic nitrogen mustard derivative, is an alkylating agent. Chlorambucil interferes with DNA replication and induces cellular apoptosis via the accumulation of cytosolic p53 and subsequent activation of Bax, an apoptosis promoter.
Approved indications
- Chronic lymphoid leukemia, disease
- Follicular non-Hodgkin's lymphoma
- Hodgkin's disease
Boxed warnings
- WARNING LEUKERAN (chlorambucil) can severely suppress bone marrow function. Chlorambucil is a carcinogen in humans. Chlorambucil is probably mutagenic and teratogenic in humans. Chlorambucil produces human infertility (see WARNINGS and PRECAUTIONS).
Common side effects
- Bone marrow suppression
- Nausea
- Vomiting
- Diarrhea
- Oral ulceration
- Tremors
- Muscular twitching
- Myoclonia
- Confusion
- Agitation
- Ataxia
- Flaccid paresis
Key clinical trials
- A Study of the Combination of Ibrutinib Plus Venetoclax Versus Chlorambucil Plus Obinutuzumab for the First-line Treatment of Participants With Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL) (PHASE3)
- Study of Acalabrutinib Versus Chlorambucil Plus Rituximab in Adult Subjects With Previously Untreated Chronic Lymphocytic Leukemia (PHASE3)
- Phase II Study of Chlorambucil and Subcutaneous Rituximab in Patients With Extranodal MALT Lymphoma (PHASE2)
- Comparison of the Treatments of Obinutuzumab + Venetoclax Versus Obinutuzumab + Chlorambucil in Patients With Chronic Lymphocytic Leukemia (PHASE3)
- Acalabrutinib, Obinutuzumab and Chlorambucil in Treatment naïve CLL (PHASE3)
- Ublituximab + TGR-1202 Compared to Obinutuzumab + Chlorambucil in Participants With Untreated and Previously Treated Chronic Lymphocytic Leukemia (PHASE3)
- Open-label Extension Study in Patients 65 Years or Older With Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma (PHASE3)
- A Global Study of Lisaftoclax (APG-2575) Combined With Acalabrutinib Versus Immunochemotherapy for Newly Diagnosed CLL/SLL. (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Leukeran CI brief — competitive landscape report
- Leukeran updates RSS · CI watch RSS
- Waylis portfolio CI