Busulfan

Treats chronic myelogenous (myeloid, myelocytic, granulocytic) leukemia. Sold as Myleran, Busulfex. Every FDA application for this active ingredient: 2 branded and 15 generic.

As of Nov 14, 2026, FDA lists 17 applications for Busulfan (sold as Myleran, Busulfex), first approved Jun 26, 1954, with 1 new use approved and 0 published complete response letters.

First approved: Jun 26, 1954
Applications: 17
New uses approved: 1
Complete response letters: 0
Medicare spending, 2025: $262K
First approved in the EU: Jul 9, 2003

Medicare Spending

CMS Part D and Part B, 2025, before manufacturer rebates; matched by generic name


Myleran	Part D	$200K	+13%	0
Busulfan	Part B	$62K	+10%	103

Orphan Designations (FDA)

5 designations, 1 approved for the designated use; see all orphan designations

Preparative therapy for pediatric patients undergoing bone marrow transplantationSuperGen, Inc. · designated Nov 25, 2002
Intrathecal therapy for neoplastic meningitisSuperGen, Inc. · designated Mar 5, 2001
Treatment of primary brain malignancies.SuperGen, Inc. · designated Jul 7, 1997
As preparative therapy in the treatment of malignancies with bone marrow transplantation.Otsuka Pharmaceutical Company, Ltd · designated Jul 28, 1994 · approved Feb 4, 1999
For use as preparative therapy for malignancies treated with bone marrow transplantation.Sparta Pharmaceuticals, Inc. · designated Apr 21, 1994

Approvals in the EU (EMA)

First approved in the EU 588 months after the US. Centrally authorised medicines, matched by ingredient; see all EU approvals

Busulfan Fresenius Kabi, genericFresenius Kabi Deutschland GmbH · Sep 22, 2014
Busilvex, new substancePierre Fabre Medicament · Jul 9, 2003 · withdrawn

Approvals in Japan (PMDA)

PMDA's English list since 2008, matched by ingredient; see all Japanese approvals

BusulfexA drug with a new dosage indicated for the conditioning treatments prior to allogeneic hematopoietic stem cell transplantation, and autologous hematopoietic… · Otsuka Pharmaceutical Co., Ltd. · Aug 25, 2021
BusulfexA drug with a new indication and a new dosage for the conditioning treatment of malignant lymphoma in patients prior to autologous hematopoietic stem- cell… · Otsuka Pharmaceutical Co., Ltd. · Mar 25, 2020
BusulfexA drug with a new additional once-daily dosage indicated for a conditioning regimen prior to allogeneic hematopoietic stem cell transplantation, and… · Otsuka Pharmaceutical Co., Ltd. · Sep 21, 2018

Clinical Trials

168 open or ongoing studies name this ingredient as an intervention, including as a comparator; newest first. See all


Busulfan-Based Versus TBI-Based Conditioning for Adult ALL-CR2 Allo-HSCTAcute Lymphoblastic Leukemia, Hematopoietic Stem Cell Transplantation · Peking University People's Hospital	Not applicable	Not yet recruiting	Nov 5, 2026
MAC vs RIC for Transplantation in FLT3-ITD-Mutated AML Guided by UHS-NGS MRD ( FLT3 - MAC vs RIC )ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION, Acute Myeloid Leukemia · First Affiliated Hospital of Zhejiang University	Not applicable	Not yet recruiting	Oct 29, 2026
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA Receiving sUCBTAplastic Anaemia · Anhui Provincial Hospital	Phase 2	Not yet recruiting	Oct 21, 2026
26DT052; Scaling Busulfan Dose to Body Surface Area in ChildrenBusulfan Pharmacokinetic Analysis, Busulfan Dosing · Children's Hospital of Philadelphia	Phase 1	Not yet recruiting	Aug 28, 2026
Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIMWHIM, Warts · National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1 / Phase 2	Enrolling by invitation	Aug 20, 2026
A Study of VABu Conditioning Regimen in Allo-HSCT for AML PatientsLeukemia, Myeloid, Acute(AML), Hematopoietic Stem Cell Transplantation (HSCT) · The First Affiliated Hospital of Soochow University	Not applicable	Not yet recruiting	Aug 13, 2026
Allogeneic ORCA-Q Stem Cell Transplant for the Treatment of Relapsed and Refractory High Risk Multiple MyelomaRecurrent Multiple Myeloma, Refractory Multiple Myeloma · University of California, Davis	Phase 1	Not yet recruiting	Aug 12, 2026
Arsenic Trioxide to Prevent Relapse in Patients Undergoing Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia or Myelodysplastic SyndromeAcute Myeloid Leukemia, Myelodysplastic Syndrome · University of Michigan Rogel Cancer Center	Phase 1	Recruiting	Jul 30, 2026

Applications

NDAs, BLAs and generic ANDAs for this ingredient, oldest first; each opens its full history


Jun 26, 1954	MyleranNDA 9386 · New molecular entity	Waylis Therap	18	Marketed
Feb 4, 1999	BusulfexNDA 20954 · New dosage form	Otsuka Pharm	16	Marketed
Dec 22, 2015	BusulfanANDA 202259	Am Regent	3	Discontinued
Mar 24, 2017	BusulfanANDA 207050	Pharmascience Inc	3	Marketed
Nov 20, 2017	BusulfanANDA 208536	Mylan Institutional	2	Discontinued
Dec 8, 2017	BusulfanANDA 205139	Actavis	3	Discontinued
Dec 18, 2017	BusulfanANDA 209580	Amneal	3	Marketed
Jul 13, 2018	BusulfanANDA 205184	Pharmobedient	1	Discontinued
Jul 31, 2018	BusulfanANDA 205672	Hospira	2	Marketed
Sep 21, 2018	BusulfanANDA 205106	Quagen	1	Discontinued
Jan 14, 2019	BusulfanANDA 207794	Nexus	0	Discontinued
Feb 22, 2019	BusulfanANDA 210148	Accord Hlthcare Inc	2	Marketed
Apr 18, 2019	BusulfanANDA 210931	Adaptis	1	Marketed
May 7, 2019	BusulfanANDA 210448	Apotex	1	Discontinued
Oct 23, 2020	BusulfanANDA 212127	Meitheal	0	Marketed
Jun 25, 2024	BusulfanANDA 215102	Eugia Pharma	0	Discontinued
Sep 11, 2024	BusulfanANDA 215235	Prinston	0	Marketed

Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA