Rare Disease Report

Potential IPF Therapy Granted Orphan Drug Designation

DECEMBER 20, 2017
Mathew Shanley
Prometic Life Sciences Inc. received its second orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of idiopathic pulmonary fibrosis (IPF) earlier this week when the status was granted to Plasminogen (Ryplazim).

The company received its first orphan drug designation for the IPF patient population in February 2015, when the FDA granted the status to the antifibrotic drug PBI-4050.

IPF is a progressive, life-threatening disease with poor prognosis. The replacement of small air sacs of the lung with fibrotic tissue cause the gradual worsening of dyspnea, and the condition is characterized by common symptoms like shortness of breath; fatigue; dry, hacking cough; and shallow breathing.

Plasminogen has been reported to perform favorably when compared to these recently-approved drugs when tested in gold-standard animal models proven to mimic pulmonary fibrosis in humans. The drug significantly reduced tissue scarring in the lungs, indicating the potential for providing clinically significant improvement and stabilization in lung function.

"The fibrinolytic systems play a central role in wound healing and tissue repair, a process believed to be abnormal within the IPF affected lung," explained Dr. John Moran, Chief Medical Officer of Prometic in a press release. "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."

Previously, standard-of-care was limited to oxygen therapy and pulmonary rehabilitation, and the only real cure is lung transplant. The 2014 approvals of Esbriet (pirfenidone) and Ofev (nintedinib) changed the landscape for treatment options, however. The term “idiopathic” is used in the name of the disease because the cause of pulmonary fibrosis is still unknown.

"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,” said Pierre Laurin, President and Chief Executive Officer of 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."

In October, the FDA announced its acceptance of the company’s Biologics License Application (BLA), granted priority review status and set a Prescription Drug User Fee Act (PDUFA) action date of April 14, 2018 for the drug as it pertains to the rare condition congenital plasminogen deficiency. Additionally, the drug was granted Rare Pediatric Disease Designation for that same indication in August.

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