PUR1900 from Pulmatrix, which is used for treating pulmonary fungal infections in patients with cystic fibrosis (CF), has received orphan drug status from the FDA.
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.