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.

openfdaRegulatory4 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	Multiple Myeloma	Multiple Myeloma-Palliative Treatment IVRA is indicated for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. IVRA is an alkylating drug indicated for palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. ( 1 )	approved	Aug 18, 2023	openfda
US FDA	Multiple Myeloma	Evomela is an alkylating drug indicated for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma.	approved	Mar 10, 2016	openfda
US FDA	Multiple Myeloma	Evomela is an alkylating drug indicated for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma.	approved	Mar 10, 2016	openfda
US FDA

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 15 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 18 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