It’s the second time the FDA has designated a Prometic IPF treatment an orphan drug. The first covered PBI-4050, a treatment for lung tissue scarring.
Only drugs or biological treatments for rare diseases can obtain orphan drug status. The FDA defines a rare disease as one that affects fewer than 200,000 Americans. An orphan designation provides several benefits to the drug’s developer, including tax incentives and potential market exclusivity for seven years after approval.
Animal studies have confirmed that the process of breaking down blood clots — or fibrinolysis — is compromised in IPF patients’ lungs. This process is vital to both wound healing and tissue repair.
Ryplazim consists of pasminogen, a protein that is part of the fibrinolytic system.
Studies have shown that low plasminogen levels lead to deposits of fibrosis-promoting material in the lungs, making breathing difficult.
Prometic researchers showed that Ryplazim significantly reduced scar tissue in the lungs of animals with pulmonary fibrosis, compared with non-treated animals. Importantly, the results were similar to those that approved IPF drugs produced.
“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,” Pierre Laurin, Prometic’s president and chief executive officer, said in a press release. “This designation supports our decision to aggressively pursue the development of Plasminogen in additional acute-care medical conditions where the healing and fibrinolysis [blood-clot-dissolving] process is impaired.”
The FDA is reviewing Prometic’s Biologic License Application for Ryplazim. The application is a request that the agency approve a therapy made by biotechnology.
Meanwhile, Prometic is starting Phase 2/3 clinical trials of PBI-4050.