Promedior, a clinical stage biotechnology company developing novel therapeutics for the treatment of fibrosis, has announced that the US Food and Drug Administration (US FDA) has granted fast track designation to PRM-151 for the treatment of myelofibrosis (MF), a serious, life-limiting cancer characterised by fibrosis of the bone marrow. This fast track designation covers primary myelofibrosis, post-polycythemia vera myelofibrosis, and post-essential thrombocythemia myelofibrosis.
The FDA grants fast track designation to a product that is intended to treat a serious condition and that has demonstrated the potential to address an unmet medical need. The advantages of fast track designation include actions to expedite development and FDA review including opportunities for frequent interactions with the FDA review team and eligibility for priority review depending on clinical data at the time of biologics license application submission. PRM-151 was awarded orphan drug designation for myelofibrosis in September 2014.
“We are extremely pleased to received fast track designation for PRM-151 as we believe that PRM-151 offers the potential to better meet the needs of patients with myelofibrosis," said Beth Tréhu, managing director, FACP, chief medical officer of Promedior. “This designation validates our perspective that there is a clear and compelling need for a novel mechanism for the treatment of myelofibrosis that specifically targets the underlying fibrotic processes of the disease. We will continue to work expeditiously to advance this programme through the clinic and look forward to presenting the full data set from the first stage of our phase 2 study later this year.”
Myelofibrosis affects approximately 18,000 people per year in the US, with a median age of 61-66. The only potentially curative treatment is allogeneic bone marrow transplant, which results in reversal of fibrosis and normalisation of blood counts, but is a realistic option for only a small number of patients. Other currently available therapies have minimal, if any, impact on the underlying fibrosis, and often result in worsening in hemoglobin and platelets, important blood parameters which are directly linked to morbidity and mortality and remain the major unmet need in patients with myelofibrosis. 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.