China’s Center for Drug Evaluation (CDE) website has indicated that Swiss pharmaceutical giant Novartis’ (NYSE: NVS) iptacopan is likely to receive priority review status for the treatment of adult paroxysmal nocturnal hemoglobinuria (PNH). This potential award highlights the fact that iptacopan is a clinically urgently needed innovative drug addressing a rare disease and has been granted breakthrough therapy designation (BTD).
Iptacopan: A First-in-Class Complement Factor B (FB) Inhibitor
Iptacopan, a first-in-class complement factor B (FB) inhibitor, is currently undergoing clinical studies for PNH and various kidney diseases with severe unmet needs involving the complement system. These include IgA nephropathy, complement 3 glomerulopathy (C3G), atypical hemolytic uremic syndrome, and membranous nephropathy. The drug’s innovative approach to targeting the complement system offers hope for patients with these rare and serious conditions.
Breakthrough Therapy Designation and Orphan Drug Designations
Iptacopan previously obtained breakthrough therapy designation in China in March 2022, emphasizing its potential to significantly improve treatment outcomes for PNH. Additionally, the drug has earned orphan drug designations (ODDs) in the US and Europe for use in PNH and C3G, further recognizing the drug’s importance in addressing rare diseases with limited treatment options.-Fineline Info & Tech
