The nomacopan clinical data set consists of over 35 cumulative years of patient safety data, with no reported drug-related serious adverse events, and a positive clinical response across five treated clinical conditions:
- Bullous Pemphigoid (subcutaneous): an autoimmune blistering disease of the elderly
- Thrombotic microangiopathy post bone marrow transplant: pediatric (subcutaneous), a severe multi-organ inflammatory disease with an 80% mortality rate in the more severe patient group under current standard of care
- COVID-19 pneumonia (subcutaneous): life-threatening disease in 5-10% of patients with COVID-19
- Atopic Keratoconjunctivitis (topical): severe surface of the eye inflammatory condition
- Paroxysmal nocturnal hemoglobinuria (subcutaneous): severe hematological condition resulting in lysis of patients’ red blood cells
- Eighteen PNH patients treated
- First patient successfully treated and self-administering for over four years
All these diseases except PNH have no approved treatment and inadequate current standard of care often consisting of broad range immunosuppressants such as steroids.
