Busulfan

Alkylating Drug — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Busulfan is an ofp drug in the Alkylating Drug class catalogued in DrugCentral. It was first approved by the FDA in 1954. Its mechanism of action is classified as Alkylating Activity. DrugCentral lists 1 molecular target, including MMP9. Recorded indications include Allogeneic bone marrow transplantation, Autologous hematopoietic stem-cell transplantation for Ewing's sarcoma family of tumors, Malignant lymphoma and Neuroblastoma.

• Approval status: ofp (first approved 1954 by the FDA).
• Drug class: Alkylating Drug.
• Mechanism of action: Alkylating Activity.
• Molecular targets: MMP9 (1 listed in DrugCentral).
• Indications: Allogeneic bone marrow transplantation, Autologous hematopoietic stem-cell transplantation for Ewing's sarcoma family of tumors, Malignant lymphoma, Neuroblastoma and Philadelphia Chromosome Positive Chronic Myelocytic Leukemia.
• ATC code: L01AB01 (Alkyl sulfonates).
• Molecular formula: C6H14O6S2; molecular weight 246.29 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (5)

• Allogeneic bone marrow transplantation
• Autologous hematopoietic stem-cell transplantation for Ewing's sarcoma family of tumors
• Malignant lymphoma
• Neuroblastoma
• Philadelphia Chromosome Positive Chronic Myelocytic Leukemia

Contraindications (18)

• Anemia
• Bacterial infectious disease
• Breastfeeding (mother)
• Epilepsy
• Fanconi's anemia
• Fibrosis of lung
• Hyperglycemia
• Hyperuricemia
• Hypokalemia
• Hypomagnesemia
• Mycosis
• Neutropenic disorder
• Portal vein obstruction
• Pregnancy, function
• Radiation oncology AND/OR radiotherapy
• Severe Bone Marrow Depression
• Thrombocytopenic disorder
• Viral disease

Mechanism & targets

• Mechanism of action (MoA): Alkylating Activity
• Established pharmacologic class: Alkylating Drug

Molecular targets (1)

Target	Gene	Action	Activity
Matrix metalloproteinase-9	MMP9	unknown	IC50 5.28

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2020-03-25	PMDA	—	Otsuka Pharmaceutical Co., Ltd
1954-06-26	FDA	—	ASPEN GLOBAL	Yes

ATC classification

ATC code	Therapeutic hierarchy
L01AB01	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › ALKYLATING AGENTS › Alkyl sulfonates

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label
• FDA label: ABL1, BCR · Informative PGx
• HCSC label

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Venoocclusive liver disease	3615.1
Acute graft versus host disease	3494.5
Graft versus host disease	3289.7
Chronic graft versus host disease	2773.7
Mucosal inflammation	2341.1
Acute graft versus host disease in skin	2305.4
Cytomegalovirus infection	2268.9
Product use in unapproved indication	2059.8
Venoocclusive disease	1930.4
Cytomegalovirus infection reactivation	1857.5
Cystitis haemorrhagic	1551.1
Graft versus host disease in skin	1255.0

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Busulfan is on its molecule page.

• Molecular formula: C₆H₁₄O₆S₂
• Molecular weight: 246.29 g/mol
• CAS number: 55-98-1
• InChIKey: COVZYZSDYWQREU-UHFFFAOYSA-N
• PubChem CID: 2478

CS(=O)(=O)OCCCCOS(C)(=O)=O

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Busulfan used for?

DrugCentral lists the following indications for Busulfan: Allogeneic bone marrow transplantation, Autologous hematopoietic stem-cell transplantation for Ewing's sarcoma family of tumors, Malignant lymphoma, Neuroblastoma and Philadelphia Chromosome Positive Chronic Myelocytic Leukemia.

How does Busulfan work?

Busulfan is classified as Alkylating Activity; its molecular targets in DrugCentral include MMP9.

When was Busulfan first approved?

Busulfan was first approved by the FDA in 1954.

What drug class does Busulfan belong to?

Busulfan is in the Alkylating Drug class.

What is the chemical formula of Busulfan?

Busulfan has the molecular formula C6H14O6S2 and a molecular weight of 246.29 g/mol.

Sources

• DrugCentral drug card 438 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL820 — compound record and bioactivity
• PubChem CID 2478 — chemical identifiers
• DrugBank DB01008 — drug record
• FDA UNII G1LN9045DK — substance identity
• DailyMed: FDA labels for Busulfan — current prescribing information (label search)
• UniProt P14780 (MMP9) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.