Melphalan is an alkylating agent used to treat multiple myeloma, ovarian carcinoma, uveal melanoma with unresectable hepatic metastases, and for high-dose conditioning before hematopoietic stem cell transplant in patients. Melphalan is a nitrogen mustard or bischloroethylamine type alkylating agent.
- Mechanism
- Curator reviewed · 9 references
Melphalan is an alkylating agent of the bischloroethylamine type. It is believed to be taken up by tumour cells via a neutral amino acid active pathway shared by leucine. Melphalan binds at the N7 position of guanine and induces inter-strand cross-links in DNA, disrupting DNA synthesis or transcription. It can also cause DNA-protein cross-linking and induce lesions in RNA, proteins, and lipids. Melphalan is cytotoxic in resting and rapidly dividing tumour cells.
- Primary indication
- Melphalan is indicated for use as a high-dose conditioning treatment prior to hematopoietic stem cell transplantation in patients with multiple myeloma.Curator reviewed · 5 structured indications
- Formula / weight
- C13H18Cl2N2O2 · 305.2 g/mol (avg)
- First approval
- Canada, 2017 · United States, 1984 · European Union, 2020
- Also known as
- 4-[bis(2-chloroethyl)amino]-L-phenylalanine · Alanine Nitrogen Mustard · L-PAM · L-Phenylalanine mustard · L-Sarcolysin
- Code names
- CB-3025 · NCI-C04853 · NSC-241286 · NSC-8806 · SK-15673 · WR-19813
- Brand names
Resolves to
SGDBTWWWUNNDEQ-LBPRGKRZSA-NSMILESN[C@@H](CC1=CC=C(C=C1)N(CCCl)CCCl)C(O)=OWhat you can answer from here — as of September 23, 2026
Which other approved drugs treat the same conditions as Melphalan, and which companies have late-stage candidates in those indications?
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