Ifenprodil for IPF Earns Orphan Drug Designation

In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. These are defined as conditions affecting fewer than 200,000 Americans. Designed to incentivize drug development within the rare disease sphere, Orphan Drug designation comes alongside benefits such as fee waivers, tax credits, and seven years of market exclusivity after approval. According to a news release from Canadian pharmaceutical development company Algernon Pharmaceuticals, Inc., the FDA recently granted Orphan Drug designation to ifenprodil, a potential therapeutic option for idiopathic pulmonary fibrosis (IPF). 

Currently, IPF comes with a relatively poor prognosis. The estimated mean survival time following diagnosis is around two to five years. There is no cure for IPF, nor any available treatment options designed to reduce or remove lung scarring. Therefore, new and effective therapeutics are urgently needed. 

Ifenprodil is an orally delivered small molecule that, according to Algernon Pharmaceuticals, was first developed to treat peripheral circulatory disorders. It is also the only active ingredient in Algernon’s NP-120, an N-methyl-d-aspartate (NDMA) receptor glutamate receptor antagonist that targets Glu2NB. Algernon is exploring this therapy for IPF, as well as for chronic cough. 

So far, the therapy has shown promise in clinical studies. For example, a Phase 2a trial found that ifenprodil was safe, effective, and well-tolerated. It noy only prevented progressive lung damage, but maintained lung function, improved cough, and enhanced quality of life (QOL). 

Idiopathic Pulmonary Fibrosis (IPF): An Overview 

Breaking down the name of idiopathic pulmonary fibrosis gives a lot of insight into the condition. Idiopathic means “of unknown cause.” Pulmonary is related to the lungs. Fibrosis refers to the thickening or hardening of tissue (in this case, scar tissue). Therefore, IPF is when lung tissue scars and hardens without a known cause. Some hypothesize that a mix of genetics and environmental triggers – such as viral infections, metal or wood dust, or cigarettes – may prompt IPF. It is rare in people under 50 years old and most often affects those in their late 60s or older.

Regardless, as the lung tissue hardens, oxygen is unable to enter the bloodstream, causing some of the characteristic symptoms. These can (but do not always) include:

  • Shortness of breath
  • A dry, hacking cough
  • Gastroesophageal reflux 
  • Appetite loss
  • Unintended weight loss
  • Clubbed fingers or toes 
  • Fatigue
  • Muscle and joint pain
  • Increased risk of pneumonia, pulmonary embolisms, and lung cancer
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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