FDA grants orphan drug designation to ifenprodil for IPF

The FDA granted orphan drug designation to ifenprodil for the treatment of patients with idiopathic pulmonary fibrosis, according to a press release from the drug’s manufacturer.

Ifenprodil (NP-120, Algernon Pharmaceuticals) is an N-methyl-d-aspartate (NMDA) receptor antagonist that prevents glutamate signaling. Along with treating IPF, ifenprodil is under development to treat chronic cough, according to the press release.

This orphan drug designation comes after Algernon Pharmaceuticals’ phase 2a study, which met both of its primary endpoints. According to the release, patients with IPF who received ifenprodil did not show worsened lung function and had significant improvements in the occurrence of cough related to IPF.

The study demonstrated that ifenprodil was safe and well-tolerated, according to the release, and it also found that both patient-reported quality of life and cough severity improved with the use of the drug.

“We appreciate the U.S. FDA’s decision to grant [orphan drug designation] status to ifenprodil for IPF, a disease for which prognosis remains dismal, with 50% mortality expected within 3 to 4 years,” Christopher J. Moreau, CEO of Algernon Pharmaceuticals, said in the release. “This regulatory milestone comes at an important time in the development of ifenprodil as a potential new therapy for IPF, as we plan the next steps for our clinical program.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.