FDA grants orphan drug designation to mocravimod for acute myeloid leukemia
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The FDA granted orphan drug designation to mocravimod for treatment of patients with acute myeloid leukemia who are undergoing allogeneic hematopoietic stem cell transplantation.
Mocravimod (KRP203, Priothera) is a novel, synthetic, sphingosine 1-phosphate receptor modulator.
Phase 1 and phase 2 trials assessed the safety and tolerability of the agent for several autoimmune indications.
Data from a phase 1B/phase 2A study of patients with hematologic malignancies prompted Priothera to further develop the agent for treatment of blood cancers, according to a company-issued press release.
The agent is designed to be an adjunctive and maintenance therapy to enhance the curative potential of HSCT for patients with AML.
A global registration-enabling phase 2B trial designed to assess mocravimod for this indication is expected to begin in the second half of this year. The study will include about 250 patients in the United States, Europe and Asia.
The European Medicines Agency (EMA) also granted orphan drug designation to mocravimod.
"The orphan drug designations we received for mocravimod from both the FDA and EMA are important milestones towards addressing the urgent, unmet needs of [patients with AML],” Florent Gros, co-founder and CEO of Priothera, said in the release. “Allogeneic stem cell transplantation is the only potentially curative approach for [patients with ALM] but has unacceptably high mortality rates with current treatments. We are looking forward to initiating the global phase 2B clinical trial with mocravimod in multiple centers in the U.S., Europe and Asia in the coming months.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
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