U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C24H30Cl2FN3O3
Molecular Weight 498.418
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of MELPHALAN FLUFENAMIDE

SMILES

CCOC(=O)[C@H](CC1=CC=C(F)C=C1)NC(=O)[C@@H](N)CC2=CC=C(C=C2)N(CCCl)CCCl

InChI

InChIKey=YQZNKYXGZSVEHI-VXKWHMMOSA-N
InChI=1S/C24H30Cl2FN3O3/c1-2-33-24(32)22(16-18-3-7-19(27)8-4-18)29-23(31)21(28)15-17-5-9-20(10-6-17)30(13-11-25)14-12-26/h3-10,21-22H,2,11-16,28H2,1H3,(H,29,31)/t21-,22-/m0/s1

HIDE SMILES / InChI

Description
Curator's Comment: description was created based on several sources, including,http://www.nhs.uk/ipgmedia/National/Macmillan%20Cancer%20Support/assets/MelphalanMCS5pages.pdf http://www.bloodjournal.org/content/bloodjournal/100/1/224.full.pdf

Melphalan, also known as L-phenylalanine mustard, phenylalanine mustard, L-PAM, or L-sarcolysin, is a phenylalanine derivative of nitrogen mustard. Melphalan is a bifunctional alkylating agent which produces a number of DNA adducts with the DNA interstrand crosslink (ICL) considered to be the critical cytotoxic lesion. Melphalan is used to treat different cancers including myeloma, melanoma and ovarian cancer.

CNS Activity

CNS Penetrant
2554
Curator's Comment: Melphalan poorly penetrates the central nervous system.

Originator

Chester Beatty Research Institute|Larionov, L.F.|Blokhin, N.
4
Curator's Comment: This substance was synthesized simultaneously both at the Chester Beatty Research Institute and in the Soviet Union.

Approval Year

1964
102
Targets

Targets

Primary TargetPharmacologyConditionPotency
Crosslinking Agent
81
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Palliative
Approved
8429
ALKERAN

Approved Use

ALKERAN Tablets are indicated for the palliative treatment of multiple myeloma and for the palliation of non-resectable epithelial carcinoma of the ovary.

Launch Date

1964
Palliative
Approved
8429
ALKERAN

Approved Use

ALKERAN Tablets are indicated for the palliative treatment of multiple myeloma and for the palliation of non-resectable epithelial carcinoma of the ovary.

Launch Date

1964
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
212 ng/mL
DRUG LABEL
https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/014691s030lbl.pdf
0.25 mg/kg single, oral
dose: 0.25 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MELPHALAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
12.84 mg × h/L
EXPERIMENT
https://www.ncbi.nlm.nih.gov/pubmed/26879446
200 mg/m² single, intravenous
dose: 200 mg/m²
route of administration: Intravenous
experiment type: SINGLE
co-administered:
MELPHALAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: UNKNOWN
498 ng × h/mL
DRUG LABEL
https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/014691s030lbl.pdf
0.25 mg/kg single, oral
dose: 0.25 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MELPHALAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
1 h
DRUG LABEL
https://www.accessdata.fda.gov/drugsatfda_docs/label/2011/014691s030lbl.pdf
0.25 mg/kg single, oral
dose: 0.25 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
MELPHALAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
70%
DRUG LABEL
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
MELPHALAN plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse events​
40 mg/m2 1 times / 3 weeks multiple, intravenous
Highest studied dose
Dose: 40 mg/m2, 1 times / 3 weeks
Route: intravenous
Route: multiple
Dose: 40 mg/m2, 1 times / 3 weeks
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
unhealthy, 18–74
n = 7
Health Status: unhealthy
Condition: Advanced cancer
Age Group: 18–74
Sex: M+F
Population Size: 7
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
DLT: Thrombocytopenia, Neutropenia...
Dose limiting toxicities:
Thrombocytopenia (grade 4, 57%)
Neutropenia (grade 4, 57%)
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
30 mg/m2 1 times / 3 weeks multiple, intravenous
MTD
Dose: 30 mg/m2, 1 times / 3 weeks
Route: intravenous
Route: multiple
Dose: 30 mg/m2, 1 times / 3 weeks
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
unhealthy, 18–74
n = 9
Health Status: unhealthy
Condition: Advanced cancer
Age Group: 18–74
Sex: M+F
Population Size: 9
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
DLT: Thrombocytopenia, Neutropenia...
Dose limiting toxicities:
Thrombocytopenia (grade 4, 44%)
Neutropenia (grade 4, 44%)
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
200 mg/m2 1 times / 70 days multiple, intravenous
MTD
Dose: 200 mg/m2, 1 times / 70 days
Route: intravenous
Route: multiple
Dose: 200 mg/m2, 1 times / 70 days
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
unhealthy, 33–65
n = 55
Health Status: unhealthy
Condition: Multiple myeloma
Age Group: 33–65
Sex: M+F
Population Size: 55
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
Disc. AE: Toxic reaction (NOS), Bacterial infection...
AEs leading to
discontinuation/dose reduction:
Toxic reaction (NOS) (grade 4, 1.8%)
Bacterial infection (grade 5, 3.6%)
Systemic herpes zoster infection (grade 5, 1.8%)
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Disc. AE: Bone marrow depression, Hypersensitivity reaction...
AEs leading to
discontinuation/dose reduction:
Bone marrow depression (severe)
Hypersensitivity reaction (2%)
Anaphylaxis
Gastrointestinal toxicity
Nausea
Vomiting
Diarrhea
Mucositis oral
Fetal damage
Infertility
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
AEs

AEs

AESignificanceDosePopulation
Neutropenia grade 4, 57%
DLT
40 mg/m2 1 times / 3 weeks multiple, intravenous
Highest studied dose
Dose: 40 mg/m2, 1 times / 3 weeks
Route: intravenous
Route: multiple
Dose: 40 mg/m2, 1 times / 3 weeks
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
unhealthy, 18–74
n = 7
Health Status: unhealthy
Condition: Advanced cancer
Age Group: 18–74
Sex: M+F
Population Size: 7
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
Thrombocytopenia grade 4, 57%
DLT
40 mg/m2 1 times / 3 weeks multiple, intravenous
Highest studied dose
Dose: 40 mg/m2, 1 times / 3 weeks
Route: intravenous
Route: multiple
Dose: 40 mg/m2, 1 times / 3 weeks
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
unhealthy, 18–74
n = 7
Health Status: unhealthy
Condition: Advanced cancer
Age Group: 18–74
Sex: M+F
Population Size: 7
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
Neutropenia grade 4, 44%
DLT
30 mg/m2 1 times / 3 weeks multiple, intravenous
MTD
Dose: 30 mg/m2, 1 times / 3 weeks
Route: intravenous
Route: multiple
Dose: 30 mg/m2, 1 times / 3 weeks
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
unhealthy, 18–74
n = 9
Health Status: unhealthy
Condition: Advanced cancer
Age Group: 18–74
Sex: M+F
Population Size: 9
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
Thrombocytopenia grade 4, 44%
DLT
30 mg/m2 1 times / 3 weeks multiple, intravenous
MTD
Dose: 30 mg/m2, 1 times / 3 weeks
Route: intravenous
Route: multiple
Dose: 30 mg/m2, 1 times / 3 weeks
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
unhealthy, 18–74
n = 9
Health Status: unhealthy
Condition: Advanced cancer
Age Group: 18–74
Sex: M+F
Population Size: 9
Sources:
https://pubmed.ncbi.nlm.nih.gov/10656432
Page: p.60
Toxic reaction (NOS) grade 4, 1.8%
Disc. AE
200 mg/m2 1 times / 70 days multiple, intravenous
MTD
Dose: 200 mg/m2, 1 times / 70 days
Route: intravenous
Route: multiple
Dose: 200 mg/m2, 1 times / 70 days
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
unhealthy, 33–65
n = 55
Health Status: unhealthy
Condition: Multiple myeloma
Age Group: 33–65
Sex: M+F
Population Size: 55
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
Systemic herpes zoster infection grade 5, 1.8%
Disc. AE
200 mg/m2 1 times / 70 days multiple, intravenous
MTD
Dose: 200 mg/m2, 1 times / 70 days
Route: intravenous
Route: multiple
Dose: 200 mg/m2, 1 times / 70 days
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
unhealthy, 33–65
n = 55
Health Status: unhealthy
Condition: Multiple myeloma
Age Group: 33–65
Sex: M+F
Population Size: 55
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
Bacterial infection grade 5, 3.6%
Disc. AE
200 mg/m2 1 times / 70 days multiple, intravenous
MTD
Dose: 200 mg/m2, 1 times / 70 days
Route: intravenous
Route: multiple
Dose: 200 mg/m2, 1 times / 70 days
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
unhealthy, 33–65
n = 55
Health Status: unhealthy
Condition: Multiple myeloma
Age Group: 33–65
Sex: M+F
Population Size: 55
Sources:
https://pubmed.ncbi.nlm.nih.gov/9720737
Page: p.248, 250
Hypersensitivity reaction 2%
Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Anaphylaxis Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Diarrhea Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Fetal damage Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Gastrointestinal toxicity Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Infertility Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Mucositis oral Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Nausea Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Vomiting Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Bone marrow depression severe
Disc. AE
100 mg/m2 1 times / day multiple, intravenous
Recommended
Dose: 100 mg/m2, 1 times / day
Route: intravenous
Route: multiple
Dose: 100 mg/m2, 1 times / day
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
unhealthy
Health Status: unhealthy
Condition: Multiple myeloma
Sources:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207155s000lbl.pdf
Page: p.1
Sourcing

Sourcing

Vendor/AggregatorIDURL
ChEBI
CHEBI:28876
https://www.ebi.ac.uk/chebi/searchId.do?chebiId=CHEBI:28876
ChemicalBook
CB2492687
https://www.chemicalbook.com/ChemicalProductProperty_EN_CB2492687
MolPort
MolPort-003-665-535
https://www.molport.com/shop/molecule-link/MolPort-003-665-535
ZINC
ZINC000000001673
http://zinc15.docking.org/substances/ZINC000000001673
eMolecules
525665
https://www.emolecules.com/cgi-bin/more?vid=525665
PubMed

PubMed

TitleDatePubMed
[Diagnosis and treatment of plasma cell leukemia].
1973 Apr 27
Carcinoma of the breast. Occurence after treatment with melphalan for multiple myeloma.
1976 Oct 4
[Refractory anemia with partial myeloblastosis in multiple myeloma treated with melphalan].
1977 Apr 9-16
Cytomegalovirus pneumonia after treatment with melphalan and prednisone. Report of a case.
1978
Drug testing using a soft agar stem cell assay on patient and xenograft tumor material.
1984 Sep
Melphalan-resistant multiple myeloma: results of treatment according to the M-2 protocol.
1985
Altered pharmacokinetics in the mechanism of chemosensitization: effects of nitroimidazoles and other chemical modifiers on the pharmacokinetics, antitumour activity and acute toxicity of selected nitrogen mustards.
1986
Cytogenetic abnormalities in acute leukemia complicating melphalan-treated multiple myeloma.
1990 Aug 1
[Hepatic veno-occlusive disease in a patient undergoing bone marrow autotransplant after busulfan and melphalan conditioning].
1990 Jan 27
Interaction of melphalan and dexamethasone in a human myeloma cell line.
1991 Dec
High-dose chemotherapy containing busulfan followed by bone marrow transplantation in 24 children with refractory or relapsed non-Hodgkin's lymphoma.
1991 Dec
Cyclophosphamide-associated carcinoma of urothelium: modalities for prevention.
1991 Nov
Multiple myeloma with coexistent myelofibrosis: improvement of myelofibrosis following recovery from multiple myeloma after treatment with melphalan and prednisolone.
1991 Sep-Oct
Veno-occlusive disease of the liver after high-dose cytoreductive therapy with busulfan and melphalan for autologous blood stem cell transplantation in multiple myeloma patients.
2007 Dec
Multicentric Castleman disease with secondary AA renal amyloidosis, nephrotic syndrome and chronic renal failure, remission after high-dose melphalan and autologous stem cell transplantation.
2007 Sep
Hypercalcemia induced by 13 cis-retinoic acid in patients with neuroblastoma.
2008 Apr
Cross-linking of the DNA repair protein Omicron6-alkylguanine DNA alkyltransferase to DNA in the presence of antitumor nitrogen mustards.
2008 Apr
Safety and efficacy of bortezomib and melphalan combination in patients with relapsed or refractory multiple myeloma: updated results of a phase 1/2 study after longer follow-up.
2008 Aug
Pharmacologic profiling of human and rat cytochrome P450 1A1 and 1A2 induction and competition.
2008 Dec
Cytogenetically defined myelodysplasia after melphalan-based autotransplantation for multiple myeloma linked to poor hematopoietic stem-cell mobilization: the Arkansas experience in more than 3,000 patients treated since 1989.
2008 Jan 1
Heterogeneity of human glioblastoma: glutathione-S-transferase and methylguanine-methyltransferase.
2008 Jul
Intralesional adenovirus-mediated interleukin-2 gene transfer for advanced solid cancers and melanoma.
2008 May
Cardiac toxicity of high-dose cyclophosphamide and melphalan in patients with multiple myeloma treated with tandem autologous hematopoietic stem cell transplantation.
2008 Sep
Common peroneal nerve palsy following TNF-based isolated limb perfusion for irresectable extremity desmoid tumor.
2009 Dec
Targeting the Fanconi anemia/BRCA pathway circumvents drug resistance in multiple myeloma.
2009 Dec 15
Development of rapid light-chain deposition disease in hepatic arteries with severe ischemic cholangitis in a multiple myeloma patient treated with melphalan, prednisone and lenalidomide.
2009 Jan
Cutaneous involvement in multiple myeloma and bortezomib.
2009 Nov
Excessive toxicity of once daily i.v. BU, melphalan and thiotepa followed by auto SCT on patients with non-Hodgkin's lymphoma.
2010 Apr
Efficacy and safety of once-weekly bortezomib in multiple myeloma patients.
2010 Dec 2
Uptake of 3-[125I]iodo-alpha-methyl-L-tyrosine into colon cancer DLD-1 cells: characterization and inhibitory effect of natural amino acids and amino acid-like drugs.
2010 Feb
Thalidomide-induced phrenic nerve paralysis.
2010 Feb
Glutathione S-transferase M1 inhibits dexamethasone-induced apoptosis in association with the suppression of Bim through dual mechanisms in a lymphoblastic leukemia cell line.
2010 Mar
Mechanisms of peripheral neuropathy associated with bortezomib and vincristine in patients with newly diagnosed multiple myeloma: a prospective analysis of data from the HOVON-65/GMMG-HD4 trial.
2010 Nov
Inhalation of alkylating mustard causes long-term T cell-dependent inflammation in airways and growth of connective tissue.
2011 Feb 27
Risk factors for, and reversibility of, peripheral neuropathy associated with bortezomib-melphalan-prednisone in newly diagnosed patients with multiple myeloma: subanalysis of the phase 3 VISTA study.
2011 Jan
Bone marrow osteoblast damage by chemotherapeutic agents.
2012
Efficient monitoring of in vivo pig-a gene mutation and chromosomal damage: summary of 7 published studies and results from 11 new reference compounds.
2012 Dec
In vitro and in vivo antitumor activity of a novel alkylating agent, melphalan-flufenamide, against multiple myeloma cells.
2013 Jun 1
The novel alkylating prodrug melflufen (J1) inhibits angiogenesis in vitro and in vivo.
2013 Oct 1
FDA-approved drugs and other compounds tested as inhibitors of human glutathione transferase P1-1.
2013 Sep 5
Repairing of N-mustard derivative BO-1055 induced DNA damage requires NER, HR, and MGMT-dependent DNA repair mechanisms.
2015 Sep 22
In vitro and in vivo activity of melflufen (J1)in lymphoma.
2016 Apr 4
In vitro and in vivo anti-leukemic activity of the peptidase-potentiated alkylator melflufen in acute myeloid leukemia.
2017 Jan 24
Melflufen - a peptidase-potentiated alkylating agent in clinical trials.
2017 Sep 12
Patents

Patents

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8
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28
JP2007531525A
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18
JP2008501654A
33
JP2008508853A
10
JP2008510829A
14
JP2008533018A
15
JP2008533019A
34
JP2008539832A
82
WO2014065751A1
4

Sample Use Guides

In Vivo Use Guide
Curator's Comment: IV dose is 16 mg/m2
Phase 3 clinical study (OCEAN, OP-103). Melflufen 40 mg i.v. on Day 1 and dexamethasone 40 mg on Days 1, 8, 15 and 22 of each 28-day cycle.
Route of Administration: Intravenous
In Vitro Use Guide
Curator's Comment: RPMI-8226 (multiple myeloma) cells were treated with 10 uM or 100 uM of melphalan.
Melflufen showed activity with cytotoxic IC50-values in the submicromolar range (0.011-0.92 μM) in the cell lines. In the primary cultures melflufen yielded slightly lower IC50-values (2.7 nM to 0.55 μM). Treated cell lines exhibited a clear accumulation in the G2/M-phase of the cell cycle.
Name Type Language
MELPHALAN FLUFENAMIDE
INN   WHO-DD  
INN   USAN  
Official Name English
L-PHENYLALANINE, 4-(BIS(2-CHLOROETHYL)AMINO)-L-PHENYLALANYL-4-FLUORO-, ETHYL ESTER
Systematic Name English
PRODRUG J1
Code English
J-1
Code English
Melphalan flufenamide [WHO-DD]
Common Name English
J1
Code English
MFF
Common Name English
melphalan flufenamide [INN]
Common Name English
J1 (PRODRUG)
Code English
J-1 (PRODRUG)
Code English
MELPHALAN FLUFENAMIDE [USAN]
Common Name English
ETHYL (2S)-2-((2S)-2-AMINO-3-(4-(BIS(2-CHLOROETHYL)AMINO)PHENYL)PROPANAMIDO)-3-(4-FLUOROPHENYL)PROPANOATE
Systematic Name English
PRODRUG J-1
Code English
Classification Tree Code System Code
NCI_THESAURUS C697
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
NCI_THESAURUS C1742
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
EU-Orphan Drug EU/3/15/1463
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
Code System Code Type Description
EPA CompTox
DTXSID40191461
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
PUBCHEM
9935639
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
RXCUI
2531369
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
WIKIPEDIA
Melphalan flufenamide
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
SMS_ID
100000177159
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
USAN
GH-114
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
FDA UNII
F70C5K4786
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
NCI_THESAURUS
C107680
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
CAS
380449-51-4
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
INN
9493
Created by admin on Fri Dec 15 20:15:37 GMT 2023 , Edited by admin on Fri Dec 15 20:15:37 GMT 2023
PRIMARY
ACTIVE MOIETY
Structure of MELPHALAN
NIH
NCATS
PRIVACY ACT
ACCESSIBILITY
DISCLAIMER
HHS VULNERABILITY DISCLOSURE
For language access assistance, contact the NCATS Public Information Officer
National Center for Advancing Translational Sciences (NCATS),
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