Medexus Pharmaceuticals announced that the FDA has extended the review period for the New Drug Application (NDA) for treosulfan by three months, with a new PDUFA target action date of January 30, 2025. This extension is to allow the FDA additional time to review supplemental analyses of previously submitted data provided by medac, the licensor of Medexus's commercialization rights to treosulfan.
The FDA has determined that the additional information constitutes a major amendment, thus requiring more time to complete the review. Importantly, the FDA has not requested the submission of new clinical data.
Ken d'Entremont, Medexus's Chief Executive Officer, stated, "We recognize that this development further extends the regulatory review process timeline. Nevertheless, we are encouraged to see that the FDA remains actively engaged with medac, and we continue to prepare for an approval of treosulfan in the United States and a commercial launch in the first half of calendar year 2025."
The treosulfan NDA is seeking approval for the use of treosulfan in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adult and pediatric patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). AML and MDS are severe hematologic malignancies that often require stem cell transplantation for potential cure. Treosulfan, if approved, would provide another option for patients undergoing this procedure.
Potential Impact of Treosulfan
Treosulfan's potential approval addresses a critical need in the treatment landscape for patients with AML and MDS undergoing allo-HSCT. The drug's unique mechanism and demonstrated efficacy could offer a valuable alternative in preparative regimens. The extension of the review period underscores the FDA's commitment to a thorough evaluation of the data, ensuring the safety and efficacy of treosulfan for this vulnerable patient population.
