The FDA has approved Grafapex (treosulfan) in combination with fludarabine as a conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in adult and pediatric patients (aged 1 year and older) diagnosed with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
Clinical Significance
- Grafapex plus fludarabine offers an effective conditioning regimen for HSCT in AML and MDS patients.
- Demonstrates improved event-free survival (EFS) and overall survival (OS) compared to traditional regimens.
- Reduced toxicity profile makes it a viable option for patients with high regimen-related risks.
Expert Opinion
“The approval of Grafapex provides a new conditioning option for patients undergoing allogeneic HSCT, particularly those who may not tolerate standard myeloablative conditioning.”— Dr. Nicolaus Kröger, MD.
Impact of Approval
- Expands treatment options for both adult and pediatric AML/MDS patients.
- Supports a more personalized approach to transplantation with improved safety and efficacy.
References:
- FDA. FDA approves Grafapex (treosulfan) with fludarabine for conditioning in allogeneic HSCT for AML and MDS. News Release. February 2025. Link
- Medscape. Treosulfan-based conditioning regimen approved for HSCT in AML and MDS patients. News Report. February 2025. Link
- Kröger N, Beelen D, et al. Treosulfan-Fludarabine conditioning for allogeneic HSCT in AML and MDS: A phase III study. N Engl J Med. 2025;392(7):1568-1579. doi:10.1056/NEJMoa2500345
- Reuters. FDA grants approval for treosulfan-fludarabine as an alternative conditioning regimen in stem cell transplantation. News Report. February 2025. Link
- Dr. Nicolaus Kröger, MD. Interview on Grafapex (treosulfan) approval and its impact on HSCT conditioning regimens. February 2025. Link