Grafapex FDA Approval History
Last updated by Judith Stewart, BPharm on Jan 28, 2025.
FDA Approved: Yes (First approved January 21, 2025)
Brand name: Grafapex
Generic name: treosulfan
Dosage form: Lyophilized Powder for Injection
Company: Medexus Pharmaceuticals Inc.
Treatment for: Stem Cell Transplant Conditioning
Grafapex (treosulfan) is an alkylating agent indicated in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation in adult and pediatric patients one year of age and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
- Allogeneic hematopoietic stem cell transplantation (alloHSCT) involves the use of stem cells from a donor. A conditioning treatment is administered to the patient before receiving alloHSCT to destroy any remaining cancer cells in the body and to weaken the patient's own immune system so that the donor cells are not rejected.
- Grafapex is a DNA alkylating agent that works by interfering with DNA replication and RNA transcription, and disrupting nucleic acid function to cause cell death.
- FDA approval was supported by data from the MC-FludT.14/L Trial II (NCT00822393) which compared treosulfan plus fludarabine (n=280) to busulfan plus fludarabine (n=290) as a preparative regimen for alloHCT. Results showed that the treosulfan regimen led to a 33% reduction in the risk of death in the overall population compared with the busulfan regimen (HR, 0.67; 95% CI, 0.51-0.90), and 27% in patients with AML (HR, 0.73; 95% CI, 0.51-1.06) and 36% in patients with MDS (HR, 0.64; 95% CI, 0.40-1.02).
- Grafapex is administered as a two hour intravenous infusion, given on three consecutive days (day -4, -3, -2) in conjunction with fludarabine before hematopoietic stem cell infusion (day 0).
- Warnings and precautions associated with Grafapex include seizures, skin disorders, injection site reactions and local tissue necrosis, increased risk of a secondary malignancy, and embryo-fetal toxicity.
- Common adverse reactions (≥20%) include musculoskeletal pain, stomatitis, pyrexia, nausea, edema, infection, and vomiting.
Selected Grade 3 or 4 nonhematological laboratory abnormalities include increased GGT, increased bilirubin, increased ALT, increased AST, and increased creatinine.
Development timeline for Grafapex
| Date | Article |
|---|---|
| Jan 23, 2025 | Approval FDA Approves Grafapex (treosulfan) Preparative Regimen for Allogeneic Hematopoietic Stem Cell Transplantation |
| Jul 25, 2022 | Treosulfan NDA Resubmitted to FDA |
| Jun 6, 2022 | Treosulfan Pivotal Study Results Published |
| Apr 22, 2022 | Medexus Pharmaceuticals Announces Resubmission of Treosulfan NDA |
| Aug 3, 2021 | Complete Response Letter Received from FDA for Treosulfan |
Further information
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