Priothera Ltd., a Phase 3 clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound mocravimod, announced that the FDA granted Orphan Drug designation (ODD) to mocravimod for the ‘treatment to improve outcome following hematopoietic stem cell transplantation in hematologic malignancies’. This ODD aims to potentially increase leukemia-free survival by enhancing a graft-versus-leukemia (GvL) response.
Florent Gros, Co-Founder and CEO of Priothera, commented: “We are very pleased that the US FDA granted mocravimod this Orphan Drug designation. This designation emphasizes the importance of developing novel therapeutic options to improve the outcome and success of maintenance therapy following allo-HSCT in blood cancer patients. This is an important milestone as this ODD complements the first ODD granted for prevention of graft-versus-host disease.
The first ODD granted for mocravimod by the US FDA was for the ‘prevention of graft-versus-host disease (GvHD)’ – see press release here.
Mocravimod, a sphingosine-1-phosphate (S1P) receptor modulator, is being investigated in a pivotal global Phase 3 study - MO-TRANS (NCT05429632) - evaluating the efficacy and safety of mocravimod as an adjunctive and maintenance therapy to allo-HSCT. The study which is enrolling approximately 250 adult Acute Myeloid Leukemia (AML) patients, is ongoing in the US, Europe, Southeast Asia and Latin America.
Mocravimod, which has been previously tested in multiple autoimmune indications, is being developed to enhance the curative potential of allo-HSCT. Moreover, it has shown a clinically meaningful outcome in a Phase 1b/2a study1 in patients with hematologic malignancies undergoing allo-HSCT.
The Orphan Drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!