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  • FDA Approval: Grafapex (Treosulfan) for Allogeneic Stem Cell Transplantation in adult and pediatric patients aged one year and older with AML or MDS

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:

  1. FDA. FDA approves Grafapex (treosulfan) with fludarabine for conditioning in allogeneic HSCT for AML and MDS. News Release. February 2025. Link
  2. Medscape. Treosulfan-based conditioning regimen approved for HSCT in AML and MDS patients. News Report. February 2025. Link
  3. 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
  4. Reuters. FDA grants approval for treosulfan-fludarabine as an alternative conditioning regimen in stem cell transplantation. News Report. February 2025. Link
  5. Dr. Nicolaus Kröger, MD. Interview on Grafapex (treosulfan) approval and its impact on HSCT conditioning regimens. February 2025. Link

 

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