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Busulfan · Myleran, NSC-750

Small molecule · Cytotoxic · developed by Waylis Therap

Approved FDA, Jun 1954

1 Registered trials

What is Busulfan?

Busulfan is a synthetic dimethane-sulfonate small molecule that acts as an alkylating agent, forming carbonium ions that cause DNA alkylation, breaks, and inhibition of DNA replication and RNA transcription.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Myleran, NSC-750, Busulphan
Drug type: Small molecule
Mechanism: Cytotoxic
Pathways: DNA alkylation, DNA replication inhibition, RNA transcription inhibition
Therapy area: Oncology
Developer: Waylis Therap
In trials of: JAZZ
FDA status: Approved as Myleran (June 26, 1954), NDA009386 · FDA label
UNII: G1LN9045DK

✅ FDA-approved uses

Chronic myelogenous leukemia

From the indications section of the FDA label (version of Jan 8, 2025). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT04217278	Phase 2/3	Acute Myeloid Leukaemia	Active not recruiting	JAZZ

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Acute Myeloid LeukaemiaHigh-risk Myelodysplastic Syndrome

Sources

Updated Oct 3, 2026.

Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z