LAVAL, QC – The U.S. Food and Drug Administration (FDA) has granted Prometic Life Science’s orally active, anti-fibrotic, lead drug candidate, PBI-4050 with orphan drug designation status as a treatment for Alstrӧm Syndrome (AS).
Alstrӧm Syndrome is a chronically debilitating condition that can lead to permanent blindness, deafness, type 2 diabetes and progressive organ failure. To this day, no satisfactory method of treatment has been authorized in the U.S. for patients affected by AS.
Prometic is currently investigating the effects of PBI-4050 on multiple organs in AS patients in an ongoing, open label, Phase 2, clinical study in the United Kingdom (UK) with plans to expand the clinical program, both in the U.S. and elsewhere in Europe, once an optimal regulatory pathway has been defined with the FDA and the European Medicines Agency, respectively.
“The positive results in patients with AS complement those we have observed in both our other trials in idiopathic pulmonary fibrosis and metabolic syndrome with type 2 diabetes, since AS includes features of both those conditions,” Dr. John Moran, Prometic’s chief medical officer commented. “AS leads to severe fibrosis in vital organs, including the heart, liver, lungs, and kidneys, with progressive multi-organ failure, and PBI-4050 has successfully reversed fibrosis in all those organs in numerous preclinical studies,” he adds.
According to Pierre Laurin, CEO of Prometic, the company’s next step will be to meet with the regulatory agencies to define the approval pathway.
Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user fees.