FDA grants Pulmatrix orphan drug status for PUR1900

Pulmatrix Inc. this week announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status for PUR1900, the company’s inhaled drug designed to treat pulmonary fungal infections in patients with cystic fibrosis (CF).

"This designation is a major boost to our efforts to make this drug available as quickly as possible to cystic fibrosis patients who currently suffer from fungal infections in their lungs, and from the allergic reactions they experience because of the fungal infections," Robert Clarke, CEO of Pulmatrix, said. "The estimated addressable market for improved antifungal treatments for CF is in the tens of thousands of patients per year, but the inhaled drug could also find much larger markets treating pulmonary fungal infections and other immunocompromised patients that could expand the addressable market to millions of patients per year."

The FDA informed Pulmatrix that its designation was “based on a plausible hypothesis that your drug may be clinically superior to the same drug that is already approved for the same indication."

Orphan drug status is granted to promising drugs that treat rare diseases or conditions, providing a quick regulatory path to market and financial incentives for companies developing the drugs.

"Our technology delivers the drug directly to the lungs," David L. Hava, CSO of Pulmatrix said. "That significantly reduces the risks of side effects and drug-drug interactions, bringing great benefits to patients."