GRAFAPEX (treosulfan)
GRAFAPEX is indicated for the treatment of Acute Myeloid Leukemia; Myelodysplastic Syndrome.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2025-01-21
- Patent Cliff
- 2026
- Routes
- INTRAVENOUS
- Dosage Forms
- POWDER
GRAFAPEX Approval History
What GRAFAPEX Treats
2 indicationsGRAFAPEX is approved for 2 conditions since its original approval in 2025. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acute Myeloid Leukemia
- Myelodysplastic Syndrome
GRAFAPEX Boxed Warning
MYELOSUPPRESSION GRAFAPEX causes severe and prolonged myelosuppression at the recommended dosage. Hematopoietic stem cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression. Monitor hematologic laboratory parameters [see Warnings and Precautions ( 5.1 )] . WARNING: MYELOSUPPRESSION See full prescribing information for complete boxed warning. GRAFAPEX causes severe and prolonged myelosuppression. Hematopoietic stem cell transplantation is requ...
WARNING: MYELOSUPPRESSION GRAFAPEX causes severe and prolonged myelosuppression at the recommended dosage. Hematopoietic stem cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression. Monitor hematologic laboratory parameters [see Warnings and Precautions ( 5.1 )] . WARNING: MYELOSUPPRESSION See full prescribing information for complete boxed warning. GRAFAPEX causes severe and prolonged myelosuppression. Hematopoietic stem cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression. Monitor hematologic laboratory parameters. ( 5.1 )
GRAFAPEX Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in GRAFAPEX's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications GRAFAPEX treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to GRAFAPEX
3 of 20FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
18 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07493538 | 2025LS162 | Ph 2 | recruiting | MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases |
| NCT03980769 | RG1005072 NCI-2019-03243, 9621 | Ph 2 | recruiting | Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders |
| NCT04965597 | BMTCTN1904 2U10HL069294-11, RG1121820 | Ph 2 | completed | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) |
| NCT04195633 | RG1005742 NCI-2019-07697, 10343 | Ph 2 | recruiting | Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies |
| NCT04994808 | RG1121616 NCI-2021-07474, 10598 | Ph 2 | active not recruiting | Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia |
| NCT02349906 | MC-FludT.16/NM | Ph 2 | completed | Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases |
| NCT05534620 | MC-FludT.19/PK | Ph 1 | completed | Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics |
| NCT05636787 TreoMel | TreoMel Trial | Ph 2 | recruiting | Clinical Trial Investigating the Chemotherapeutic Compound Treosulfan (Trecondi® Ideogen) in Myeloma Patients |
| NCT01949129 | ALL SCTped FORUM 2012 | Ph 2, Ph 3 | recruiting | Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia |
| NCT06198842 | Treo-NBS 2023 | Ph 2 | recruiting | Low Dose Treosulfan Based Conditioning Regimen and PTCy in HSCT for Nijmegen Breakage Syndrome |
| NCT03961919 FLAT-Auto | 2008-000664-16 | Ph 2 | completed | Phase II Trial in Elderly Patients With AML or MDS in Complete Remission Not Eligible for Allogenic Transplant |
| NCT00796068 results posted | 2275.00 NCI-2010-00299, 2275 | Ph 2 | completed | Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation in Treating Patients With Hematological Cancer Who Are Undergoing Umbilical Cord Blood Transplant |
| NCT00919503 results posted | 2256.00 NCI-2010-01277, 2256.00 | Ph 2 | completed | Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders |
| NCT00860574 results posted | 2272.00 NCI-2010-00315, P01HL036444 | Ph 2 | completed | Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia |
| NCT00822393 | MC-FludT.14/L EudraCT-No.: 2008-002356-18 | Ph 3 | completed | Clinical Phase III Trial Treosulfan-based Conditioning Versus Reduced-intensity Conditioning (RIC) |
| NCT02333058 | MC-FludT.17/M | Ph 2 | completed | Treosulfan-based Conditioning in Paediatric Patients With Haematological Malignancies |
| NCT00987636 EWING2008 | 108128 2008-003658-13 | Ph 3 | completed | Study in Localized and Disseminated Ewing Sarcoma |
| NCT01772953 | 11-TREO | Ph 2 | completed | Treosulfan/Fludarabine/Low Dose TBI as a Preparative Regimen for Children With AML/MDS Undergoing Allo HCT |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
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.
GRAFAPEX FDA Label Details
Indications & Usage
FDA Label (PDF)GRAFAPEX is indicated for the treatment of Acute Myeloid Leukemia; Myelodysplastic Syndrome.
WARNING: MYELOSUPPRESSION GRAFAPEX causes severe and prolonged myelosuppression at the recommended dosage. Hematopoietic stem cell transplantation is required to prevent potentially fatal complications of the prolonged myelosuppression. Monitor hematologic laboratory parameters [see Warnings and Pre...
GRAFAPEX Patents & Exclusivity
Patents (1 active)
Exclusivity
Pro Intelligence Preview
Deep insights for GRAFAPEX
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2026
- • 4 active patents
Trial Analysis
- • 18 total trials
- • Stage: Stable
Competitive Landscape
- • 20 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment