Alvelestat for A1AD Earns Orphan Drug Status

According to a late October 2021 news release from biopharmaceutical company Mereo BioPharma Group plc (“Mereo”), the company’s therapeutic candidate alvelestat received Orphan Drug designation from the FDA. The therapy is designed to treat people with alpha-1 antitrypsin deficiency (A1AD), with a specific focus on A1AD-related lung disease. Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases (those affecting under 200,000 Americans). Because alvelestat has received this status, Mereo also earns a variety of benefits, from fee waivers and tax credits to 7 years market exclusivity upon approval.


So what exactly is alvelestat? According to Mereo, alvelestat is:

an oral neutrophil elastase inhibitor.

While Mereo does not believe that the treatment can assist with liver damage, the company does believe that alvelestat has the potential to halt or prevent further lung damage in patients. Over 1,100 people have been dosed with alvelestat in prior studies. So far, the therapy has been shown to be relatively safe and well-tolerated. Mereo is also currently conducting a Phase 2 clinical trial evaluating alvelestat in 165 patients with A1AD.

Alpha-1 Antitrypsin Deficiency (A1AD)

SERPINA 1 gene mutations cause alpha-1 antitrypsin deficiency (A1AD), an inherited condition characterized by low Alpha-1 antitrypsin levels. Normally, SERPINA 1 plays a role in producing this protein, which protects the body against the neutrophil elastase (NE) enzyme. Without enough alpha-1 antitrypsin, NE can attack bodily tissues, causing issues in the lung and liver. Those of European heritage are at a higher risk of having A1AD. Symptoms vary, but usually appear between ages 20-50. These include:

  • Shortness of breath and/or difficulty breathing
  • Wheezing
  • Unintended weight loss
  • Chronic cough
  • Jaundice (yellowing of the skin and eyes)
  • Recurrent respiratory infections
  • Exercise or activity intolerance
  • Rapid heart rate upon standing
  • Fatigue
  • Dark urine
  • Emphysema
  • A harsh, hacking cough
  • Swollen hands and feet
  • Pruritus (intense and chronic itching)
  • Barrel-shaped chest
  • Skin lesions
  • Cirrhosis
  • Liver disease
  • Panniculitus
  • Abdominal distention
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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