ProMetic Life Sciences Inc. is currently preparing an Orphan Drug Designation (ODD) request for the company’s novel Idiopathic Pulmonary Fibrosis (IPF) drug PBI-4050 to the Food and Drug Administration (FDA).
According to the company, an external panel of IPF world experts strongly recommend that PBI-4050 be granted orphan drug status in order to treat the disease after reviewing the drug’s extensive anti-fibrotic preclinical and phase I safety data. “Our preclinical data has demonstrated robust improvements in pathology and breadth of response in key bio-markers implicated in the progression of this deadly disease,” said Mr. Pierre Laurin, President and Chief Executive Officer of ProMetic in a company press release. “We are very enthusiastic about the potential of PBI-4050 to offer breakthrough therapy clinical benefits to patients suffering from this deadly medical condition.”
Prometic is initiating the IPF clinical program in Canada this quarter, and plans to include other identified clinical test venues in the United Kingdom and eventually the United States.
Dr. John Moran, ProMetic’s Chief Medical officer, explained that “the ability of PBI-4050 to reduce or improve some of the most recognized inflammatory and fibrotic indicators, typically representative of lesions and scars in the lungs, provides a compelling case to aggressively investigate the use of this oral therapy to treat IPF patients.”
PBI-4050 was recently compared to Pirfenidone, a commercially approved therapy for medical use to treat IPF, in a standard animal model designed to emulate IPF in humans, revealing that PBI-4050 significantly reduced tissue scarring in the lungs. Furthermore, PBI-4050 combined with Pirfenidone reduced fibrotic markers, which indicates the potential for improvement and stabilization of lung function. The PBI-4050 phase I clinical trial was deemed a success, with the 40 volunteers involved in the study revealing that the drug is safe and well-tolerated.
IPF is a chronic and fatal disease in which lungs progressively decline. The small air sacs of the lungs, the alveoli, become replaced by fibrotic tissue (scars) and causes worsening dyspnea and breathlessness. The cause of the disease remains unknown, and it is associated with a poor prognosis. Usually, it occurs in adults between 50 and 70 years old, both men and women are affected, and it is closely related with those who have smoked. IPF affects about 130,000 people in the U.S., and almost 50,000 new cases are diagnosed each year. 40,000 die annually from IPF.