According to an August 2022 news release from biotechnology company Elixiron Immunotherapeutics (“Elixiron”), EI-1071, a therapy for patients with idiopathic pulmonary fibrosis (IPF), recently earned Orphan Drug designation from the FDA.
Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare conditions. In the United States, a “rare” condition is one affecting fewer than 200,000 individuals. Drug developers – in this case, Elixiron – also earn benefits such as fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval.
Currently, EI-1071 has been explored in Phase 1 and preclinical trials. Researchers have shared that EI-1071, a small molecule CSF-1R inhibitor, has shown promise in safety, efficacy, and tolerability. For example, EI-1071 could improve respiratory function, reduce inflammation, and help prevent lung scarring. Outside of IPF, Elixiron is also developing EI-1071 as a potential therapeutic option for individuals with Alzheimer’s disease.
What is Idiopathic Pulmonary Fibrosis (IPF)?
Idiopathic pulmonary fibrosis (IPF) is aptly named: the hardening or scarring (fibrosis) of lung tissue (pulmonary) due to an unknown cause (idiopathic). This chronic lung disease inhibits adequate oxygen from entering into the bloodstream, causing a number of health issues. An estimated 13 to 20 out of every 100,000 people have idiopathic pulmonary fibrosis. Although there is no known cause, some researchers and doctors hypothesize that genetic predispositions and environmental triggers could lead to IPF. Risk factors include being older in age, being male, smoking cigarettes, and having a family history of lung disease. Symptoms can (but do not always) include:
- A chronic dry, hacking cough
- Shortness of breath and/or difficulty breathing
- Gastroesophageal reflux (GERD)
- Fatigue
- Joint and muscle pain
- Appetite loss
- Unintended weight loss
- Clubbed fingers and toes
- Increased risk of lung cancer, pneumonia, or pulmonary embolism
