ProMetic Life Sciences, Inc., a biopharmaceutical company focused on plasma-derived therapeutics and small-molecule drug development, recently announced that its product PBI-4050 for the treatment of idiopathic pulmonary fibrosis (IPF) has been granted orphan drug designation status by the European Commission.
IPF is a progressive fatal lung disease of unknown cause in which the alveoli and the lung tissue become thick and scarred (fibrosis), leading to severe breathing difficulties, shortness of breath, and a compromised oxygen transfer between the lungs and the bloodstream. There is no cure for IPF and it is estimated that 128,100 individuals in the United States suffer from the disease. IPF has a poor prognosis and the five-year mortality rate is estimated to be 50% to 70%. Effective therapies for pulmonary fibrosis are therefore urgently needed.
PBI-4050 is an oral drug candidate designed to treat fibrosis, a process characterized by the replacement of normal tissue by fibrotic scar tissue and which can impair the function of vital organs such as the lungs, heart, kidneys, and liver. PBI-4050 has been shown to be safe, well-tolerated and to significantly reduce lung tissue scarring in animal models.
PBI-4050 is currently being evaluated in an open-label Phase II clinical trial in 40 IPF patients in terms of safety, tolerability, and efficacy on lung function, disease progression, and fibrotic/inflammatory biomarkers.
“This is the second orphan drug designation secured by ProMetic regarding PBI-4050 for the treatment of IPF following the similar grant by the US FDA in February 2015. This grant confirms that there is still an urgent need in Europe for a safe and effective treatment of this devastating disease,” noted ProMetic’s President and Chief Executive Officer, Pierre Laurin, in a press release about PBI-4050.
The European Orphan Drug Designation is granted to new drugs for the treatment of rare conditions that affect less than 250,000 individuals in the European Union. This designation offers to the drug developer a 10-year period of marketing exclusivity (after the drug’s approval), reduced fees for regulatory activities, and the possibility to apply for marketing authorization in the European Union and protocol assistance (scientific advice for orphan medicines).
ProMetic is also planning to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in the beginning of 2016 for a double-blind, multicenter, placebo-controlled pivotal trial on PBI-4050 with IPF patients under therapy with nintedanib or pirfenidone, two anti-fibrotic drugs indicated for IPF treatment.