Drug · Small Molecule

Melphalan

CI-DRUG-00000373Explore in graph →NCIt C633 CHEMBL852 CIViC Approved

Regulatory

Approvals (13)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

health-canada-dpdRegulatory13 approval records from 3 sources (hover a row badge for its dataset) · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
CA Health Canada	—	Marketed in Canada as TARO-MELPHALAN (DIN 02480026) under ATC L01AA03 MELPHALAN. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed	Jan 25, 2021	health-canada-dpd
CA Health Canada	—	Marketed in Canada as MELPHALAN FOR INJECTION (DIN 02468921) under ATC L01AA03 MELPHALAN. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	withdrawnsource: Dormantsince Apr 13, 2023	Nov 20, 2018	health-canada-dpd
CA Health Canada	—	Marketed in Canada as MELPHALAN FOR INJECTION (DIN 02473348) under ATC L01AA03 MELPHALAN. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed

Health Canada records are DIN-level: one row per marketed product (brand, strength, form). The Drug Product Database does not publish indications, so no cancer is stated for these rows, and a cancelled or dormant DIN is the status of that one product — not a withdrawal of the molecule.

Data updated 16 hours agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	80	38	82	571	13	CA, EU, US	Dec 31, 1964	1990-07
Non-Hodgkin Lymphoma	Approved	Phase 4	31	11	32	230	4	US	Jun 9, 2009	1994-01
Multiple Myeloma	Approved	Phase 4	21	7	23	170	4	US	Jun 9, 2009	1994-01
Leukemia	Phase3	Phase 3	32	15	8	148	0	—

Curated evidence

Clinical evidence (25)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–25 of 25 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
FANCC Loss-of-function1
Chlorambucil + Cisplatin

Clinical trials

Trials with this intervention (578)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 551–578 of 578 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT00003662	Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic Diseases	completed	Phase 2	90	Roswell Park Cancer InstituteOTHER	1	Mar 7, 2011	clinicaltrials
NCT00055653	Donor Umbilical Cord Blood Transplantation in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders	completed

Molecule

Record

Kind: Small Molecule
Mechanism: DNA inhibitor
UNII: 1VXP4V453T
Aliases: 4-[bis(2-chloroethyl)amino]-L-phenylalanine, Alanine Nitrogen Mustard, Alkeran, CB 3025, CB-3025, EVOMELA, IVRA, L-PAM, L-Phenylalanine Mustard, L-Sarcolysin, L-Sarcolysine, L-Sarcolysin Phenylalanine Mustard, Melfalan, Melphalan, Melphalan For Injection-Hepatic Delivery System, Melphalan Hydrochloride, MELPHALAN HYDROCHLORIDE, Melphalanum, NCI-C04853, NSC-241286, NSC-8806, P-di(chloroethyl)amino-L-phenylalanine, Phelinun, Phenylalanine Mustard, Phenylalanine Nitrogen Mustard, Sarcoclorin, Sarcolycin, l-, Sarkolysin, SK 15673, TARO-MELPHALAN … (+1)

Data updated 19 hours agoSource updated unknown

Identifiers

Codes (18)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01AA03	MELPHALAN
DIN (Health Canada)	00004715

KRAS G12A6
Melphalan	Multiple Myeloma	Predictive	B	Supports Resistance	2	accepted	EID2009 In a study of patients receiving melphalan-based therapy, those with KRAS codon 12 mutations were less likely to have a positive response than those with wildtype KRAS (26.9% vs. 58.3%, P=0.015). PMID 19284554 · Greco et al., 2009 · Open in CIViC	civic
〃	Multiple Myeloma	Predictive	B	Supports Resistance	—	submitted	EID2010 Activating mutations in KRAS or NRAS were observed more frequently in multiple myeloma patients than in patients with monoclonal gammopathy of uncertain significance (MGUS) (31% vs. 5% ). Another stu… (full text at CIViC) PMID 11524732 · Bezieau et al., 2001 · Open in CIViC	civic
〃	Multiple Myeloma	Predictive	B	Supports Resistance	—	submitted	EID2011 Activating mutations in KRAS or NRAS were observed more frequently in multiple myeloma patients than in patients with monoclonal gammopathy of uncertain significance (MGUS) (31% vs. 5% ). Another stu… (full text at CIViC) PMID 15339850 · Rasmussen et al., 2005 · Open in CIViC	civic
〃	Multiple Myeloma	Predictive	D	Supports Resistance	2	accepted	EID2012 In in vitro experiments, myeloma cell lines expressing activating NRAS and KRAS mutations are less sensitive to the apoptosis-inducing effects of dexamethasone, doxorubicin, and melphalan in compariso… (full text at CIViC) PMID 11050000 · Rowley et al., 2000 · Open in CIViC	civic
〃	Multiple Myeloma	Predictive	D	Supports Resistance	2	accepted	EID2013 In in vitro experiments, myeloma cell lines expressing activating NRAS and KRAS mutations are less sensitive to the apoptosis-inducing effects of dexamethasone, doxorubicin, and melphalan in compariso… (full text at CIViC) PMID 12483530 · Rowley et al., 2002 · Open in CIViC	civic
〃	Multiple Myeloma	Predictive	D	Supports Resistance	2	accepted	EID2014 In in vitro experiments, myeloma cell lines expressing activating NRAS and KRAS mutations are less sensitive to the apoptosis-inducing effects of dexamethasone, doxorubicin, and melphalan in compariso… (full text at CIViC) PMID 16497971 · Hoang et al., 2006 · Open in CIViC	civic