announced that the United States Food and Drug Administration (U.S. FDA) has granted Orphan Drug Designation (ODD) to Ifenprodil as a treatment for Idiopathic Pulmonary Fibrosis (IPF). Ifenprodil is the sole active ingredient in NP-120, an NMDA receptor antagonist and the Company’s lead clinical candidate being developed for the treatment of IPF and chronic cough.
Algernon Pharmaceuticals recently concluded a Phase 2a study of Ifenprodil in patients with IPF. The trial met its co-primary IPF endpoint with patients receiving Ifenprodil experiencing no worsening of their lung function, and significant improvements were seen in the frequency of their IPF-associated cough as well. In addition, improvements in patient-reported measures of cough severity and quality of life were observed. Ifenprodil was also confirmed to be safe and well-tolerated in the study.
Supporting the development of and evaluation of new treatments for rare diseases through ODD is a priority for the U.S FDA and other jurisdictions including Europe, that have similar orphan programs. The designation is available only for rare diseases, defined by the U.S. FDA as those which affect fewer than 200,000 patients in the United States. ODD qualifies sponsors for incentives including tax credits for qualified clinical trials and a da exemption from user fees.
If a product receiving ODD is subsequently granted approval, it receives seven years of market exclusivity, meaning that the U.S FDA may not approve any other applications, including a full New Drug Application, for the same product in the same indication.
Christopher J. Moreau, CEO of Algernon made some comments:
We appreciate the U.S. FDA’s decision to grant ODD status to Ifenprodil for IPF, a disease for which prognosis remains dismal, with 50% mortality expected within 3-4 years. 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.
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