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.

emaRegulatory1 approval record · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
EU EMA	—	High-dose of Phelinun used alone or in combination with other cytotoxic medicinal products and/or total body irradiation is indicated in the treatment of: multiple myeloma, malignant lymphoma (Hodgkin, non-Hodgkin lymphoma), acute lymphoblastic and myeloblastic leukemia, childhood neuroblastoma, ovarian cancer, mammary adenocarcinoma. Phelinun in combination with other cytotoxic medicinal products is indicated as reduced intensity conditioning (RIC) treatment prior to allogeneic haematopoietic stem cell transplantation (allo-HSCT) in malignant haematological diseases in adults. Phelinun in combination with other cytotoxic medicinal products is indicated as conditioning regimen prior to allogeneic haematopoietic stem cell transplantation in haematological diseases in the paediatric population as: Myeloablative conditioning (MAC) treatment in case of malignant haematological diseases RIC treatment in case of non-malignant haematological diseases.	approved	Nov 16, 2020	ema

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 101–150 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
NCT00112827	Melphalan and Radiation Therapy Followed By Lenalidomide in Treating Patients Who Are Undergoing Autologous Stem Cell Transplant for Stage I, Stage II, or Stage III Multiple Myeloma	completed	Phase 1 / Phase 2	54	City of Hope Medical CenterOTHER	1	Jun 6, 2025	clinicaltrials
NCT01350258	Bone Marrow Transplant Using a Reduced Intensity Regimen That is Given in Two Steps

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