Pharma Focus Europe

FDA Grants Orphan Drug Designation to Mocravimod for Allogeneic HSCT in Hematologic Malignancies - Priothera

Monday, November 27, 2023

Priothera Ltd. has announced that the US Food and Drug Administration (FDA) granted Orphan Drug designation (ODD) to its compound mocravimod, a S1P receptor modulator. This designation is for the treatment aimed at improving outcomes following hematopoietic stem cell transplantation in hematologic malignancies, with the potential to enhance leukemia-free survival by boosting a graft-versus-leukemia (GvL) response.

Florent Gros, Co-Founder and CEO of Priothera, expressed satisfaction with the FDA's decision, highlighting the significance of developing innovative therapeutic options to improve maintenance therapy outcomes after allo-HSCT in blood cancer patients. This ODD complements the initial designation granted for the prevention of graft-versus-host disease (GvHD).

Mocravimod, known as a sphingosine-1-phosphate (S1P) receptor modulator, is currently undergoing investigation in a pivotal global Phase 3 study (MO-TRANS, NCT05429632) evaluating its efficacy and safety as an adjunctive and maintenance therapy to allo-HSCT. The study, involving approximately 250 adult Acute Myeloid Leukemia (AML) patients, is ongoing in the US, Europe, Southeast Asia, and Latin America.

The dual mode of action of mocravimod, highlighted by the two ODDs, aims to improve allo-HSCT treatment outcomes by potentially increasing leukemia-free survival through a graft-versus-leukemia response while reducing tissue damage resulting from graft-versus-host disease. The Orphan Drug designation underscores the importance of mocravimod in treating rare, life-threatening, or chronically debilitating diseases.

Mocravimod, previously tested in multiple autoimmune indications, has shown promising results in a Phase 1b/2a study in patients with hematologic malignancies undergoing allo-HSCT. The drug is being developed to enhance the curative potential of allo-HSCT and improve outcomes for AML patients, addressing a high unmet medical need in the field.



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