Busulfan
Orphan DrugFDA Approved
Description
Busulfan is an alkylating agent used as a conditioning regimen prior to hematopoietic stem cell transplantation. It is commonly employed in the treatment of severe combined immunodeficiency disorders requiring bone marrow transplantation. The drug works by destroying bone marrow cells to prepare the patient for transplant.
Indications & Therapeutic Use
Conditioning treatment prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia, severe combined immunodeficiency
Global Availability (5 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Busulfan
| Generic Name | Busulfan |
| Brands | 1 brand available |
| Active Ingredient | Busulfan |
| Drug Class | Conditioning treatment prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia |
| Manufacturer | Otsuka Pharmaceutical |
| Dosage Forms | IV infusion, 6mg/mL; Oral tablet, 2mg |
| Medical Code | L01AB01 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT00000000 |
| Countries | 5 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations6 Validated Nodes