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-dpdRegulatory8 approval records · 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 18 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 1–50 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
NCT06377540	MT2022-60: Ph 2 Study of Pembro+ BEAM With ASCT for Relapsed Hodgkin Lymphoma	recruiting	Phase 2	28	Masonic Cancer Center, University of MinnesotaOTHER	1	Sep 29, 2026	clinicaltrials
NCT07605416	Testing an Experimental Approach to Treat Patients With Plasma Cell Leukemia, The QUANTUM Trial	recruiting	Phase 2

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 21 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

KRAS G12C6
Melphalan	Multiple Myeloma	Predictive	B	Supports Resistance	2	rejected	EID2258 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	EID2259 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	EID2260 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	EID2261 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	—	accepted	EID2262 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	EID2263 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

KRAS G12D6
Melphalan	Multiple Myeloma	Predictive	B	Supports Resistance	2	rejected	EID2247 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	EID2248 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	EID2249 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	EID2250 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	—	accepted	EID2251 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	EID2252 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

KRAS G12S6
Melphalan	Multiple Myeloma	Predictive	B	Supports Resistance	2	rejected	EID2274 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	EID2275 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	EID2276 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	EID2277 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	EID2278 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	EID2279 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