ProMetic Life Sciences Inc., a biopharmaceutical company developing bioseparations, plasma-derived therapeutics and small-molecule drugs, recently announced that the European Commission has granted orphan drug designation status to the company’s drug candidate PBI-4050 to treat patients with a diagnosis of idiopathic pulmonary fibrosis (“IPF”).
Currently, the company is examining the tolerability, safety and effects of PBI-4050 on disease progression, pulmonary function, and inflammatory/fibrotic biomarkers in an open-label Phase II clinical trial in Canada in 40 patients with IPF. The company is expecting to file an IND with the FDA during the first trimester of 2016 for a pivotal, multi-center, double-blind, placebo-controlled trial in patients with IPF currently being treated with nintedanib or pirfenidone, who will be randomly assigned to receive treatment either with PBI-4050 or a placebo.
Commenting on the European orphan drug designation grant, Mr. Pierre Laurin, ProMetic’s President and Chief Executive Officer, said: “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.”
Idiopathic pulmonary fibrosis (IPF) is a chronic, devastating, and ultimately fatal disease characterized by a progressive decline in lung function. It is a specific type of interstitial lung disease in which the small air sacs of the lung, the “alveoli,” gradually become replaced by fibrotic (scar) tissue and is the cause of worsening dyspnea (shortness of breath). The cause of the disease is unknown, limited to the lungs and associated with a histopathologic and/or radiologic pattern of usual interstitial pneumonia.
PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles confirmed in several in vivo experiments targeting fibrosis in comparison to recently approved drugs for the treatment of the disease. The drug was found to reduce lung tissue scarring in non-treated animals, demonstrating the potential for stabilization and improvement of lung function in IPF patients. A formulation of PBI-4050 and another approved drug agent demonstrated a further reduction in fibrotic biomarkers in this IPF animal model, indicating that a synergic clinical benefit may be established.
European Orphan Drug Designation is granted to novel drugs or biologics that treat a rare disease or condition affecting fewer than 250,000 patients in the European Union. Sponsors who obtain orphan designation benefit from a number of incentives, including protocol assistance, a type of scientific advice specific to designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available depending on the status of the sponsor and the type of service required.