Prometic Life Sciences Inc., a company based out of Laval, Quebec, announces that an orphan drug designation status has been granted to its Plasminogen (Ryplazim) for the treatment of idiopathic pulmonary fibrosis by the US Food and Drug Administration (FDA).
In a gold-standard animal model proven to emulate pulmonary fibrosis in humans, Prometic’s Plasminogen performed favourably compared to other recently approved IPF drugs to treat this condition. Plasminogen significantly reduced tissue scarring in the lungs that was observed in non-treated animals, signifying the potential for providing clinically significant improvement and stabilization in lung function.
Idiopathic pulmonary fibrosis 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, gradually become replaced by fibrotic (scar) tissue and is the cause of worsening shortness of breath. IPF is usually associated with a poor prognosis. Approximately 40,000 people with IPF die each year, a similar number of deaths to those due to breast cancer. The 5-year mortality rate for patients with IPF is estimated to range from 50 to 70 per cent, and those with acute exacerbation have a mortality rate as high as 85 per cent with a mean survival period of a few months.
“The fibrinolytic systems play a central role in wound healing and tissue repair, a process believed to be abnormal within the IPF affected lung,” says Dr. John Moran, chief medical officer, Prometic. “Animal models of pulmonary fibrosis have demonstrated an imbalance between thrombosis and fibrinolysis within the alveolar compartment, a finding that is also observed in IPF patients. We plan to evaluate whether Plasminogen can help lung function of IPF patients during acute exacerbation episodes which would be both complementary to anti-fibrotic chronic therapy and addressing and unmet medical need in the IPF patient population.”
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.
“We are pleased to have secured a second IPF orphan drug designation from the FDA with our Plasminogen, Ryplazim, for the treatment of this devastating disease following the initial orphan drug designation received for PBI-4050, our small molecule,” says Pierre Laurin, president and chief executive officer, Prometic. “This designation supports our decision to aggressively pursue the development of Plasminogen in additional acute care medical conditions where the healing and fibrinolysis process is impaired.”