FDA Grants Orphan Drug Status to Prometic’s Pulmonary Fibrosis Therapy Ryplazim

The U.S. Food and Drug Administration has granted orphan drug status to Prometic Life Sciences’ Ryplazim (plasminogen) as a treatment for idiopathic pulmonary fibrosis.

Fewer than 200,000 Americans have IPF, which makes it eligible for orphan status. The designation gives companies incentives to develop drugs for the small markets typical of rare diseases. These include tax credits for therapy research costs, clinical trial design assistance, and being able to market an approved treatment exclusively for seven years.

Ryplazim is a protein called plasminogen that the body converts into the enzyme plasmin, which is crucial to wound healing.

Prometic believes Ryplazim can reduce IPF flare-ups. It was as effective as FDA-approved drugs in animal studies. Ryplazim-treated animals had significantly less lung tissue scarring than untreated animals.

Prometic plans clinical trials to see what benefits Ryplazim offers IPF patients, including  stabilizing their lung function during flare-ups.

The company is also planning a Phase 2/3 trial for another IPF drug, PBI-4050, which proved effective in a Phase 2 trial (NCT02538536).

“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 fibrinolytic systems play a central role in wound healing and tissue repair, a process believed to be abnormal within the IPF-affected lung,” said John Moran, Prometic’s chief medical officer. “We plan to evaluate whether Plasminogen (Ryplazim) can help lung function of IPF patients during acute exacerbation episodes which would be both complementary to anti-fibrotic chronic therapy and addressing an unmet medical need in the IPF patient population.”