Orphan Drug Status Sought for NP-120 to Treat IPF With Cough
Algernon will also file pre-investigational new drug application for study next year
Algernon Pharmaceuticals has filed for orphan drug status for NP-120 (ifenprodil), an experimental oral medicine for patients with idiopathic pulmonary fibrosis (IPF) who have persistent, hard-to-treat cough.
“The FDA has a standard response time of 90 days to requests for orphan designation, and so we should know by the end of the year whether it has been granted,” Christopher Bryan, PhD, Algernon’s vice president of Research and Operations, said in an emailed statement to Pulmonary Fibrosis News.
Bryan noted that, “in parallel to the orphan request,” Algernon will file a pre-investigational new drug application (IND) “in order to confirm details of the company’s plan to begin a larger multinational Phase 2 study in IPF next year.” An IND is a formal request therapy developers make to regulators to ask permission to initiate a clinical trial.
The filing with the U.S. Food and Drug Administration (FDA) came after the company got full results from its Phase 2a clinical trial (NCT04318704) where NP-120 was found to be safe and well tolerated to ease cough and maintain lung function in IPF patients.
Those who entered the trial also said NP-120 helped bring about gains in quality of life related to their cough. The results encouraged the company to continue advancing NP-120’s development for IPF with cough as its key indication.
“Algernon will continue working to accelerate the development of ifenprodil as a potential new therapy for IPF with cough,” Christopher J. Moreau, Algernon’s CEO, said in a press release.
Getting the designation would make the company eligible for a number of incentives, which could help speed the medicine’s regulatory approval. These include tax credits for any upcoming clinical trials, waiving fees for filing a marketing authorization, and seven years of market exclusivity during which similar medicines for the same indication cannot be placed on the market, if it’s approved.
“Orphan designation provides a number of incentives, including lowered development costs and additional FDA guidance, both of which are enormously beneficial to a smaller company like Algernon,” Bryan said.
IPF happens when the lungs become scarred for an unknown reason, making it hard for patients to breathe. Besides having shortness of breath and feeling tired, many patients with IPF have a persistent dry cough which can get worse during certain activities and be hard to treat.
NP-120 works by blocking GluN2B, a subunit of a type of protein called N-methyl-D-aspartate (NMDA) receptor, to which glutamate normally binds. Glutamate is a neurotransmitter that conveys messages from nerve cell to nerve cell (or other types of cells). GluN2B is found in lung cells and certain immune cells called T-cells and neutrophils. By blocking it, NP-120 is expected to interfere with glutamate signaling, which is known to play a role in both fibrosis (tissue scarring) and coughing.