TheraRadar
Data updated: May 26, 2026

MYLERAN (busulfan)

Alkylating Activity
Oncology Approved 1954-06-26

MYLERAN is indicated for the treatment of Chronic Myelogenous Leukemia.

Source: FDA Label • WAYLIS THERAP
1
Indication
--
Phase 3 Trials
1
Priority Reviews
71
Years on Market

Details

Status
Prescription
First Approved
1954-06-26
Routes
ORAL
Dosage Forms
TABLET

Companies

Active Ingredient: BUSULFAN

MYLERAN Approval History

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Original
New Indication
New Form
Label Update
19 FDA actions from 1954 to 2003
Dec 2003 SUPPL
Label · Labeling
Apr 2003 SUPPL
Label · Labeling
Jan 2003 SUPPL Priority
Mfg · Manufacturing (CMC)

What MYLERAN Treats

1 indications

MYLERAN is approved for 1 conditions since its original approval in 1954. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Chronic Myelogenous Leukemia
Source: FDA Label

MYLERAN Boxed Warning

WARNING MYLERAN is a potent drug. It should not be used unless a diagnosis of chronic myelogenous leukemia has been adequately established and the responsible physician is knowledgeable in assessing response to chemotherapy. MYLERAN can induce severe bone marrow hypoplasia. Reduce or discontinue the dosage immediately at the first sign of any unusual depression of bone marrow function as reflected by an abnormal decrease in any of the formed elements of the blood. A bone marrow examination shoul...

MYLERAN Competitive Set

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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

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BUSULFEX
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OTSUKA PHARM
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THIOGUANINE
THIOGUANINE
1 shared
WAYLIS THERAP
Shared indications:
Chronic Myelogenous Leukemia
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Clinical Trial Registry

194 trials
Trial Sponsor ID Phase Status Title
NCT05564390 NCI-2022-07006 NCI-2022-07006, MYELOMATCH Ph 2 recruiting MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
NCT06954987 NCI-2025-03015 NCI-2025-03015, MM3TCT-A03 Ph 2 not yet recruiting Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)
NCT02629120 160032 16-I-0032 Ph 1, Ph 2 active not recruiting High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
NCT07524530 10002233 002233-C Ph 2 not yet recruiting Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers
NCT06996119 23822 NCI-2025-03603, 23822 Ph 1 recruiting Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant
NCT06079879 3543-006 2023-504865-21-00, IMG-7289-CTP-301 Ph 3 recruiting A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)
NCT05027945 10000404 000404-C Ph 2 recruiting A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
NCT04872595 21-193 Ph 2 active not recruiting A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant
NCT05463133 10000977 000977-I Ph 1, Ph 2 recruiting 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
NCT07566377 26-168 Ph 2 recruiting Cord Blood Transplantation in Children and Young Adults With Blood Cancer
NCT06506461 SAGES1 U01HL163983 Ph 1 recruiting Gene Editing For Sickle Cell Disease
NCT02727803 2015-0313 NCI-2016-00584, 2015-0313 Ph 2 recruiting Personalized NK Cell Therapy in CBT
NCT06158828 ABCD-NK 202401147 Ph 1, Ph 2 recruiting Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML
NCT04339777 200070 20-C-0070 Ph 2 recruiting Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
NCT07565220 HCC 26-008 Ph 1 not yet recruiting Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies
NCT07113743 10000186 000186-I Ph 1, Ph 2 enrolling by invitation Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease
NCT01306019 110007 11-I-0007 Ph 1, Ph 2 recruiting Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
NCT05327023 10000489 000489-C Ph 1, Ph 2 recruiting Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies
NCT01857934 results posted NB2012 NCI-2013-00034 Ph 2 completed Therapy for Children With Advanced Stage Neuroblastoma
NCT04859946 2020-0971 NCI-2021-02784, 2020-0971 Ph 2 active not recruiting Itacitinib for the Prevention of Graft Versus Host Disease
NCT02250937 2014-0431 NCI-2014-02324, 2014-0431 Ph 2 active not recruiting Venetoclax and Sequential Busulfan, Cladribine, and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
NCT01384513 results posted 11D.247 2011-31, JT 1795 Ph 2 completed A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
NCT04384692 RG1006507 NCI-2020-01626, 10093 Ph 2 active not recruiting Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis
NCT04547049 NFD-001 Ph 3 active not recruiting A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
NCT05735717 2021LS061 Ph 2 recruiting MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
NCT06325709 10001580 001580-I Ph 1, Ph 2 recruiting Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
NCT02251821 results posted 9033 NCI-2014-01882, 9033 Ph 2 completed JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary Myelofibrosis
NCT06959771 10002385 002385-I Ph 1, Ph 2 recruiting Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
NCT04904588 ACCESS ACCESS Ph 2 active not recruiting HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide
NCT04644016 20-480 Ph 2 recruiting Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
NCT03970096 RG1005364 9749, NCI-2019-03188 Ph 2 recruiting Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)
NCT06872333 2024LS140 Ph 2 recruiting Allo HSCT for High Risk Hemoglobinopathies
NCT06345495 2023-0899 NCI-2024-02814 Ph 2 recruiting High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
NCT05565105 2021-KOE-001 Ph 2 not yet recruiting CD34+ Transplants for Leukemia and Lymphoma
NCT05617625 2021-KOE-002 Ph 2 suspended CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome
NCT07441967 IIT2025140 Ph 2 not yet recruiting The Safety and Efficacy of the Regimen of Thiotepa + Fludarabine +Granulocyte Colony-Stimulating Factor+ Cytarabine + Busulfan in Single Unrelated Umbilical Cord Blood Transplantation
NCT03126916 ANBL1531 NCI-2016-01734, ANBL1531 Ph 3 recruiting Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
NCT05170828 PRESERVE Ph 1 withdrawn Cryopreserved MMUD BM With PTCy for Hematologic Malignancies
NCT04708054 2020-0790 NCI-2020-13919, 2020-0790 Ph 2, Ph 3 recruiting Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
NCT07228624 RG1125655 NCI-2025-07728, FHIRB0020989 Ph 2 recruiting Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes
NCT06207799 2023-0448 NCI-2024-00110 Ph 2 recruiting Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy With Elranatamab in Patients With High-risk Multiple Myeloma
NCT03619551 CSIDE PBMTC NMD1801 Ph 2 active not recruiting Conditioning SCID Infants Diagnosed Early
NCT06851767 10002273 002273-I Ph 1, Ph 2 enrolling by invitation Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
NCT02861417 2016-0137 NCI-2017-00614, 2016-0137 Ph 2 active not recruiting Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant
NCT03538899 17-22799 CLIN2-10830, CLIN2-17127 Ph 1, Ph 2 recruiting Autologous Gene Therapy for Artemis-Deficient SCID
NCT01625351 RADIANT NCI-2012-00588 Ph 1 completed A Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas
NCT06001385 OPTIMIZE OPTIMIZE Ph 2 active not recruiting HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
NCT03579875 2016LS161 MT2017-17 Ph 2 recruiting Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
NCT05917405 FLUCLORIC RC22_0524 Ph 2 recruiting Study Comparing the Efficacy of 2 RIC Regimens (Clofarabine vs Fludarabine) in Adults With AML Eligible to Allo-SCT
NCT06252870 CY-MET-RIC RC23_0286 Ph 2 recruiting Study Testing Two Conditioning Regimen With a Single Prophylaxis of GVHD by Cyclophosphamide and Methotrexate Post-transplant in Patients Eligible for Matched-donor Allograft Transplantation

Showing 50 of 194 trials

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Active Pipeline

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

MYLERAN FDA Label Details

Indications & Usage

FDA Label (PDF)

MYLERAN is indicated for the treatment of Chronic Myelogenous Leukemia.

⚠️ BOXED WARNING

WARNING MYLERAN is a potent drug. It should not be used unless a diagnosis of chronic myelogenous leukemia has been adequately established and the responsible physician is knowledgeable in assessing response to chemotherapy. MYLERAN can induce severe bone marrow hypoplasia. Reduce or discontinue the...

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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.