2022-12-07| Trials & Approvals

Algernon Wins FDA Orphan Drug Designation For Rare Lung Disease Candidate

by Joy Lin
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Following a successful Phase 2 study, Vancouver, Canada-based Algernon Pharmaceuticals has obtained Orphan Drug Designation (ODD) from the US FDA for Ifenprodil, their candidate for idiopathic pulmonary fibrosis (IPF) and chronic cough. If approved, Ifenprodil could enjoy seven years of market exclusivity, courtesy of the ODD status. 

“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,” said Christopher J. Moreau, CEO of Algernon.

“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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Treatment For Progressive Lung Condition Without Cure

There is currently no cure for IPF, a type of chronic lung condition where the tiny air sacs in the lungs become damaged and scarred. This results in a decline in lung function, making it difficult for oxygen to get into the blood. 70-85% of patients with the disease suffer from a dry non-productive cough that gets worse on exertion. Limited treatment options for IPF include pirfenidone and nintedanib, which aim to slow the progressive scarring. 

Enter Ifenprodil, a N-methyl-D-aspartate (NMDA) receptor antagonist that specifically targets the NMDA-type subunit 2B (GluN2B) to inhibit glutamate signaling. The NMDA receptor is found in many tissues including lung cells, T-cells, and neutrophils. 

In a recently concluded Phase 2a proof-of-concept study of Ifenprodil in IPF, patients treated with the drug experienced no worsening of lung function, meeting a co-primary endpoint. At 12 weeks, 13 out of 20 treated patients showed stable or better lung capacity. The drug significantly reduced the frequency of IPF-related cough while improving cough severity and quality of life. 

While the drug was confirmed to be safe and well-tolerated in the study, common side effects included digestive issues (25%) and decreased appetite (10%). 

Based on the successful topline results of the Phase 2a trial, Algernon is planning to start the process of asking the FDA to approve the launch of a Phase 2b IPF study that will evaluate a once-daily dose of Ifenprodil. Patients in the Phase 2a study took the drug three times daily. 

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