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Atlas / Drug / Melphalan

Melphalan

drug
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Also known as Alanine nitrogen mustardAlkeranCB 3025CB-3025L-pamL-phenylalanine mustardL-sarcolysinMelfalanNCI-C04853NSC-241286NSC-8806Phenylalanine mustardSarcolycinl-SK 15673SK-15673SID17389837SID26752787SID56463160

Summary

Melphalan (CHEMBL852) is an approved small-molecule antineoplastic agent (ATC L01AA03); indicated across 108 conditions including plasma cell myeloma and carcinoma; with CIViC clinical evidence for 5 variant-indication associations (e.g. KRAS G12A in multiple myeloma).

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01AA03
  • Indications: 108 conditions
  • Clinical trials: 653
  • Precision-oncology evidence (CIViC): 5 variant–indication associations
  • Chemistry: 305.2 Da · C13H18Cl2N2O2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL852
NameMelphalan
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID460612
ChEBICHEBI:28876
ATCL01AA03
Molecular formulaC13H18Cl2N2O2
Molecular weight305.2
InChIKeySGDBTWWWUNNDEQ-LBPRGKRZSA-N

SMILES: C1=CC(=CC=C1C[C@@H](C(=O)O)N)N(CCCl)CCCl

IUPAC name: (2S)-2-amino-3-[4-[bis(2-chloroethyl)amino]phenyl]propanoic acid

ChEBI definition: A phenylalanine derivative comprising L-phenylalanine having [bis(2-chloroethyl)amino group at the 4-position on the phenyl ring.

Pharmacological roles (ChEBI): antineoplastic agent, carcinogenic agent, alkylating agent, immunosuppressive agent, drug allergen.

Also known as: Alanine nitrogen mustard, Alkeran, CB 3025, CB-3025, L-pam, L-phenylalanine mustard, L-sarcolysin, Melfalan, Melphalan, NCI-C04853, NSC-241286, NSC-8806

Parent form; salt/anhydrous children: CHEMBL1200863

Patent coverage: 42,372 distinct patent families (175,967 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 18 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Survival motor neuron protein, 4’-phosphopantetheinyl transferase ffp, Alpha-2A adrenergic receptor, Muscarinic acetylcholine receptor M2, Muscarinic acetylcholine receptor M1, Acetylcholinesterase, Prostaglandin G/H synthase 1, Mu-type opioid receptor, D(3) dopamine receptor.

Bioactivity

ChEMBL activities: 6 potent at pChembl ≥ 5 of 19 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
ALDH1A15.55Potency2818nMCHEMBL_ACT_4151353
CYP3A45.5Potency3162nMCHEMBL_ACT_4965532
CYP3A45.5Potency3162nMCHEMBL_ACT_5034665
KCNH25.32AC504800nMCHEMBL_ACT_25117913
Q9F4F75.25Potency5623nMCHEMBL_ACT_4380845
PTGS15.17AC506817nMCHEMBL_ACT_25206308

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

108 indications (4 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

IndicationTrial phaseMONDOEFO
plasma cell myeloma4MONDO:0009693EFO:0001378
carcinoma4MONDO:0004993EFO:0000313
neoplasm4MONDO:0005070EFO:0000616
cutaneous melanoma3MONDO:0005012EFO:0000389
melanoma3MONDO:0005105EFO:0000756
neoplasm of mature B-cells3MONDO:0004949EFO:0000096
Ewing sarcoma3MONDO:0012817EFO:0000174
myelodysplastic syndrome3MONDO:0018881EFO:0000198
acute myeloid leukemia3MONDO:0018874EFO:0000222
diffuse large B-cell lymphoma3MONDO:0018905EFO:0000403
leukemia3MONDO:0005059EFO:0000565
lymphoma3MONDO:0005062EFO:0000574
neuroblastoma3MONDO:0005072EFO:0000621
sarcoma3MONDO:0005089EFO:0000691
smoldering plasma cell myeloma3MONDO:0005235EFO:0003073
mantle cell lymphoma3MONDO:0018876EFO:1001469
soft tissue sarcoma3MONDO:0018078EFO:1001968
ganglioneuroblastoma3MONDO:0005035EFO:0000502
breast neoplasm3MONDO:0021100MONDO:0007254
ovarian cancer3MONDO:0008170MONDO:0008170
follicular lymphoma3MONDO:0018906MONDO:0018906
retinoblastoma3MONDO:0008380MONDO:0008380
sickle cell disease3MONDO:0011382MONDO:0011382
hereditary amyloidosis3MONDO:0018634MONDO:0019438
plasma cell neoplasm3MONDO:0004959EFO:0000200
ocular melanoma3MONDO:0006325EFO:1000403
uveal melanoma3MONDO:0006486EFO:1000616
colorectal neoplasm3MONDO:0005335MONDO:0005575
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
Hodgkins lymphoma2MONDO:0004952EFO:0000183
peripheral T-cell lymphoma, not otherwise specified2MONDO:0004964EFO:0000211
acute lymphoblastic leukemia2MONDO:0004967EFO:0000220
Burkitt lymphoma2MONDO:0007243EFO:0000309
chronic myeloid leukemia2MONDO:0011996EFO:0000339
renal cell carcinoma2MONDO:0005086EFO:0000681
lymphoid neoplasm2MONDO:0005157EFO:0001642
primary myelofibrosis2MONDO:0009692EFO:0002430
medulloblastoma2MONDO:0007959EFO:0002939
relapsing-remitting multiple sclerosis2MONDO:0005314EFO:0003929
testicular cancer2MONDO:0005447EFO:0005088
hematologic disorder2MONDO:0005570EFO:0005803
non-Hodgkin lymphoma2MONDO:0018908EFO:0005952
plasma cell leukemia2MONDO:0018689EFO:0006475
male breast carcinoma2MONDO:0005628EFO:0006861
pancreatic neuroendocrine neoplasm2MONDO:0005815EFO:0007331
juvenile myelomonocytic leukemia2MONDO:0011908EFO:1000309
inflammatory breast carcinoma2MONDO:0006804EFO:1000984
acute erythroid leukemia2MONDO:0017858EFO:1001257
thalassemia2MONDO:0000984EFO:1001996
histiocytosis2MONDO:0002637HP:0100727
angioimmunoblastic T-cell lymphoma2MONDO:0004977EFO:0000255
acquired polycythemia vera2MONDO:0009891EFO:0002429
anaplastic large cell lymphoma2MONDO:0020325EFO:0003032
plasmacytoma2MONDO:0005615EFO:0006738
stiff-person syndrome2MONDO:0008491EFO:0007498
Waldenstrom macroglobulinemia2MONDO:0100280EFO:0009441
Langerhans cell histiocytosis2MONDO:0018310EFO:1000318
Sezary syndrome2MONDO:0017844EFO:1000785
mycosis fungoides2MONDO:0009691EFO:1001051
chronic myelomonocytic leukemia2MONDO:0020311EFO:1001779
aplastic anemia2MONDO:0015909HP:0001915
myeloproliferative neoplasm2MONDO:0020076EFO:0002428
brain neoplasm2MONDO:0021211EFO:0003833
neuromyelitis optica2MONDO:0019100EFO:0004256
myasthenia gravis2MONDO:0009688EFO:0004991
Guillain-Barre syndrome, familial2MONDO:0007691EFO:0009538
opsoclonus-myoclonus syndrome2MONDO:0015247EFO:1001383
bronchiectasis2MONDO:0004822HP:0002110
neuroendocrine carcinoma2MONDO:0002120MONDO:0002120
kidney cancer2MONDO:0002367MONDO:0002367
graft versus host disease2MONDO:0013730MONDO:0013730
T-cell non-Hodgkin lymphoma2MONDO:0015760MONDO:0015760
nasal cavity and paranasal sinus lethal midline granuloma2MONDO:0006828MONDO:0019472
hematopoietic and lymphoid system neoplasm2MONDO:0002334MONDO:0044881
myelodysplastic/myeloproliferative disease2MONDO:0020077MONDO:0020077
MALT lymphoma2MONDO:0007650EFO:0000191
Wiskott-Aldrich syndrome2MONDO:0010518MONDO:0010518
Diamond-Blackfan anemia2MONDO:0015253MONDO:0015253
severe combined immunodeficiency2MONDO:0015974MONDO:0015974
hemoglobinuria2MONDO:0003656MONDO:0100244
retina neoplasm2MONDO:0021231EFO:1000509
Crohn disease1MONDO:0005011EFO:0000384
anemia1MONDO:0002280EFO:0004272
beta thalassemia1MONDO:0019402Orphanet:848
anaplastic oligodendroglioma1MONDO:0016696EFO:0002501
central nervous system neoplasm1MONDO:0006130EFO:1000158
peritoneal neoplasm1MONDO:0006901MONDO:0002087
fallopian tube neoplasm1MONDO:0021092MONDO:0002158
blast phase chronic myelogenous leukemia, BCR-ABL1 positive1MONDO:0006115EFO:1000131
chordoma1MONDO:0008978MONDO:0008978
immune system disorder1MONDO:0005046EFO:0000540
hereditary breast ovarian cancer syndrome1MONDO:0003582Orphanet:145
lymphoid leukemia1MONDO:0005402EFO:0004289
glioma1MONDO:0021042MONDO:0003268
neuroepithelial neoplasm0MONDO:0021193MONDO:0021193

13 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 653.

Phase distribution

PhaseTrials
PHASE2309
PHASE1110
PHASE388
PHASE1/PHASE284
Not specified43
PHASE2/PHASE310
EARLY_PHASE16
PHASE43

Top trials by phase / activity

NCTPhaseStatusTitle
NCT03829371PHASE4ACTIVE_NOT_RECRUITINGSTUDY COMPARING TWO STANDARD TREATMENTS IN AUTOLOGOUS STEM CELL TRANSPLANTATION INELIGIBLE POPULATION AFFECTED BY MULTIPLE MYELOMA
NCT01731886PHASE4COMPLETEDLenalidomide and Dexamethasone With/Without Stem Cell Transplant in Patients With Multiple Myeloma
NCT02773550PHASE4TERMINATEDTreatment With a Scheme With Low Doses of Bortezomib / Melphalan / Prednisone (MPV) in Patients With Multiple Myeloma
NCT00114101PHASE3ACTIVE_NOT_RECRUITINGLenalidomide in Treating Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplant
NCT00602641PHASE3ACTIVE_NOT_RECRUITINGMelphalan, Prednisone, and Thalidomide or Lenalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma
NCT01704716PHASE3RECRUITINGHigh Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)
NCT02443077PHASE3ACTIVE_NOT_RECRUITINGIbrutinib Before and After Stem Cell Transplant in Treating Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma
NCT03742297PHASE3ACTIVE_NOT_RECRUITINGTreatment for Elderly Fit Newly Diagnosed Multiple Myeloma Patients Aged Between 65 and 80 Years
NCT04221035PHASE3RECRUITINGHigh-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN)
NCT04799002PHASE3RECRUITINGTopotecan and Melphalan for Retinoblastoma
NCT05022901PHASE3ACTIVE_NOT_RECRUITINGAn Open-Label Expanded Access Study of the Melphalan/Hepatic Delivery System (HDS) in Patients With Hepatic Dominant Ocular Melanoma
NCT05457556PHASE3ACTIVE_NOT_RECRUITINGMismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome
NCT05674539PHASE3ENROLLING_BY_INVITATIONReduced Intensity Conditioning Regimens for Acute Myeloid Leukemia and Myelodysplastic Syndrome
NCT05991908PHASE3RECRUITINGRandomized Study of Conditioning of Fludarabine Combined With Single or Dual Alkylating Agents in Myeloid Malignancies
NCT06022939PHASE3RECRUITINGComparing Dara-VCD Chemotherapy Plus Stem Cell Transplant to Dara-VCD Chemotherapy Alone for People Who Have Newly Diagnosed AL Amyloidosis
NCT06172296PHASE3RECRUITINGDinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
NCT06613035PHASE3NOT_YET_RECRUITINGTwice-per-weekSelinexor, 2 Days Melphalan
NCT06829472PHASE3RECRUITINGConditioning Regimen of Melphalan-busulfan and Fludarabine: a Randomized Study of Different Dose of Melphalan
NCT07396480PHASE3RECRUITINGFludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine/Melphalan Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in AML/MDS Patients Aged > 50 Years: a Multicenter, Randomized, Phase 3 Trial
NCT00001296PHASE3COMPLETEDA Randomized Phase III Trial of Hyperthermic Isolated Limb Perfusion With Melphalan, Tumor Necrosis Factor, and Interferon-Gamma in Patients With Locally Advanced Extremity Melanoma
NCT00002548PHASE3COMPLETEDSWOG-9321 Melphalan, TBI, and Transplant vs Combo Chemo in Untreated Myeloma
NCT00002556PHASE3COMPLETEDCombination Chemotherapy With or Without High Dose Cyclophosphamide and Recombinant Interferon Alfa-2b in Treating Patients With Previously Untreated Stage I-III Multiple Myeloma
NCT00002599PHASE3UNKNOWNCombination Chemotherapy and Interferon Alfa With or Without Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Myeloma
NCT00002653PHASE3UNKNOWNCombination Chemotherapy With or Without Cyclophosphamide and Prednisone in Treating Older Patients With Multiple Myeloma
NCT00002678PHASE3COMPLETEDCombination Chemotherapy in Treating Patients With Multiple Myeloma
NCT00002802PHASE3COMPLETEDTherapy Based on Stage of Disease and Risk Assessment in Treating Children With Neuroblastoma
NCT00002835PHASE3COMPLETEDCombination Chemotherapy in Treating Patients With Lymphoma
NCT00002945PHASE3COMPLETEDHigh Dose Chemotherapy, Peripheral Stem Cell Transplantation, and Interleukin-2 in Treating Patients With Acute Myeloid Leukemia
NCT00002989PHASE3UNKNOWNCombination Chemotherapy With or Without Idarubicin and Peripheral Stem Cell Transplantation in Treating Patients With Leukemia or Myelodysplastic Syndrome
NCT00003215PHASE3COMPLETEDCombination Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Newly Diagnosed Aggressive Non-Hodgkin’s Lymphoma
NCT00003578PHASE3UNKNOWNHigh Dose Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Intermediate- or High-Grade Non-Hodgkin’s Lymphoma
NCT00003603PHASE3UNKNOWNChemotherapy Plus Steroid Therapy in Treating Patients With Multiple Myeloma
NCT00003789PHASE3COMPLETEDMelphalan With or Without Tumor Necrosis Factor in Treating Patients With Locally Advanced Melanoma of the Arm or Leg
NCT00003815PHASE3UNKNOWNChemotherapy and Radiation Therapy Plus Bone Marrow Transplantation in Treating Patients With Aggressive Non-Hodgkin’s Lymphoma
NCT00003816PHASE2/PHASE3COMPLETEDCombination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
NCT00003972PHASE3COMPLETEDCombination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Stage II or Stage IIIA Breast Cancer
NCT00004165PHASE3COMPLETEDMelphalan Followed by Peripheral Stem Cell Transplantation in Treating Patients With Multiple Myeloma
NCT00004188PHASE3COMPLETEDCombination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Neuroblastoma
NCT00004921PHASE3COMPLETEDHigh-Dose Chemotherapy Compared With Standard Chemotherapy in Treating Patients With Stage III or Stage IV Ovarian Epithelial Cancer That Has Been Removed During Surgery
NCT00005589PHASE3COMPLETEDCombination Chemotherapy Plus Peripheral Stem Cell Transplantation With or Without Rituximab in Treating Patients With Relapsed Non-Hodgkin’s Lymphoma

Clinical evidence (CIViC)

Variant × indication × effect (5 predictive associations from 22 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
KRAS G12AMultiple MyelomaResistanceMelphalanCIViC BEID2009 +5
KRAS G12CMultiple MyelomaResistanceMelphalanCIViC BEID2259 +4
KRAS G12DMultiple MyelomaResistanceMelphalanCIViC BEID2248 +4
KRAS G12SMultiple MyelomaResistanceMelphalanCIViC BEID2275 +4
FANCC Loss-of-functionPancreatic CancerSensitivity/ResponseCisplatin + Melphalan + Chlorambucil + Mitomycin + GemcitabineCIViC DEID1307

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 17

This page pulls from 17 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclinical_trialsefogtopdb_interactiongtopdb_ligandhgncmeshmondopatent_compoundpharmgkbpharmgkb_guidelinepubchemuniprot