Promedior Inc., a clinical stage biotechnology company developing novel therapeutics for the treatment of fibrosis, announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for PRM-151, for the treatment of myelofibrosis, a serious, life-limiting cancer characterized by fibrosis of the bone marrow.
The FDA’s Office of Orphan Drug Products grants orphan drug designation to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 U.S. patients. Orphan drug designation provides Promedior certain benefits, including seven years of marketing exclusivity upon regulatory approval, a waiver of Prescription Drug User Fee Act (PDUFA) filing fees, the opportunity to apply for annual grant funding, clinical trial design assistance, and tax credits for clinical research costs.
“Orphan drug designation is a significant step forward in the development of PRM-151, and we are excited to continue advancing PRM-151 to better meet the needs of patients with myelofibrosis,” said Suzanne Bruhn, president and chief executive officer of Promedior. “Based on the encouraging clinical results reported to date in myelofibrosis patients we believe that PRM-151’s novel mechanism of action is compelling with its potential to target and reverse the fundamental bone marrow fibrosis that underlies patients’ disease.”
With a novel mechanism of action targeted to prevent and reverse fibrosis, PRM-151 has the potential to address the fundamental fibrotic pathology of myelofibrosis. Symptomatic myelofibrosis affects approximately 18,000 people per year in the US, with a median age of 61-66.1 The only potentially curative treatment is allogeneic bone marrow transplant, which results in reversal of fibrosis and all symptoms, but is a realistic option for only a small number of patients. Other currently available therapies address the symptoms, but have minimal, if any, impact on the underlying fibrosis.
Preliminary data from a Phase 2 study of PRM-151 demonstrated benefits across all clinically relevant measures of myelofibrosis, including decreases in bone marrow fibrosis, symptom responses, improvements in hemoglobin and platelets, and reductions in spleen size, with a well-tolerated safety profile and no treatment related myelosuppression.
Date: September 2, 2014