FDA

Grafapex OK’d for HSCT conditioning in AML, MDS patients

January 28, 2025

Brand name: Grafapex

Generic name: treosulfan

Manufacturer: Medexus Pharmaceuticals

Approval date: January 22, 2025

FDA approved Grafapex (treosulfan), an alkylating agent, with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients ≥1 year of age with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Efficacy

Approval was based on results from the phase 3 MC-FludT.14/L Trial II (NCT00822393) that compared treosulfan vs. busulfan, both given with fludarabine, in 570 patients. The primary efficacy outcome was overall survival (OS), defined as time from randomization until death from any cause. Hazard ratio for OS (stratified by donor type and risk group) compared with busulfan was 0.67 (95% confidence interval [CI], 0.51-0.90) in the randomized population, 0.73 (95% CI, 0.51-1.06) in patients with AML, and 0.64 (95% CI, 0.40-1.02) in patients with MDS.

Safety

The most common adverse events reported in the trial were musculoskeletal pain, stomatitis, pyrexia, nausea, edema, infection, and vomiting. Grade 3 or 4 non-hematologic lab abnormalities included increased GGT, bilirubin, ALT, AST, and creatinine.

Source:

Medexus announces FDA approval of Grafapex (treosulfan) for injection and provides business update. Medexus Pharmaceuticals. 2025. https://www.medexus.com/en_US/news-media/press-releases/detail/176/medexus-announces-fda-approval-of-grafapex-treosulfan-for