The designation was granted “based on a plausible hypothesis that (PUR1900) may be clinically superior to the same drug that is already approved for the same indication,” according to a press release.
Recent studies demonstrated that itraconazole taken orally is effective in the treatment of allergic reactions in CF patients with fungal lung infections. High doses are needed to receive enough of the antifungal drug in the lungs through the bloodstream but side effects including liver toxicity can be severe.
“Our technology delivers the drug directly to the lungs,” said David L. Hava, PhD, Pulmatrix’s chief scientific officer. “That significantly reduces the risks of side effects and drug-drug interactions, bringing great benefits to patients.”
Pulmatrix’s proprietary PUR1900 is an inhaled anti-fungal that combines itraconazole with the company’s dry powder iSPERSE technology that allows patients to inhale the drug easily. The iSPERSE technology aims to reduce the toxicity that results from high doses of itraconazole.
Orphan drug status is granted to promising treatments for diseases that are rare, effect less than 200,000 people in the U.S., or can help more than 200,000 people but are not expected to provide financial gain for drug companies after development and marketing costs. The designation provides financial incentives for drug companies and a faster regulatory path to market the therapies.
“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,” said Robert Clarke, PhD, Pulmatrix’s chief executive officer. “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.”