NDAINJECTIONINJECTABLEPriority Review
Approved
Feb 1999
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
20
Data completeness
4/8 — Partial
Priority Review
BUSULFEX (busulfan) by Otsuka is alkylating activity [moa]. First approved in 1999.
Drug data last refreshed 4h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
BUSULFEX (busulfan) is an intravenous alkylating agent approved in 1999 for use as a conditioning regimen prior to hematopoietic progenitor cell transplantation. It works by covalently binding to DNA and RNA, leading to strand breakage and cell death in rapidly dividing cells. The drug is primarily used in hematologic malignancies and severe aplastic anemia patients undergoing bone marrow or stem cell transplant.
LOE Approaching
Product lifecycle approaching loss of exclusivity with moderate competitive pressure (30/100), suggesting a contracting team and focus on cost management rather than growth initiatives.
Mechanism of Action
Alkylating Activity
Pharmacologic Class:
Alkylating Drug
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus
Started Nov 2025
0Severe Combined Immune Deficiency (SCID)
Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
Started Jul 2022
50 enrolled
Chronic Granulomatous Disease
Target Busulfan Exposure in Children With HSCT in China
Started Nov 2020
500 enrolled
Hematopoietic Stem Cell Transplantation
Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide
Started Oct 2019
4 enrolled
Chronic Granulomatous Disease
Busulfan, Melphalan, Escalating Carfilzomib Conditioning Auto Stem Cell Transplantation for Multiple Myeloma (MM)
Started May 2019
36 enrolled
Multiple Myeloma
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.