Priothera’s mocravimod has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of leukaemia patients undergoing a stem cell transplant. The drug aims to improve outcomes for patients with haematologic malignancies following a haematopoietic stem cell transplantation. As a result of the designation, Priothera will be eligible for tax credits for clinical trials conducted in the US and, if approved, will have seven years of market exclusivity in the designated indication. Haematopoietic stem cell transplantation is a common treatment for various blood cancers, with nearly 60,000 new cases of leukaemia in the US each year.
This is the second orphan drug designation for Priothera’s mocravimod, with the first designation granted in March 2022 for the treatment of acute myeloid leukaemia (AML) in patients undergoing hematopoietic stem cell transplantation. The drug’s dual mode of action aims to prevent graft-versus-host disease while enhancing the graft-versus-leukaemia response. Priothera’s CEO, Florent Gros, highlighted the potential of mocravimod to increase leukaemia-free survival and reduce tissue damage resulting from graft-versus-host disease. Mocravimod is currently being investigated in a Phase III trial to assess its efficacy and safety as an adjunctive and maintenance treatment for AML patients undergoing haematopoietic cell transplantation.