Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G
Posted December 16th, 2020 for Novartis Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disorder, resulting in debilitating symptoms that can impact patients’ quality of life 1–3 C3 glomerulopathy (C3G) is a rare renal disease, affecting young patients with a poor prognosis and significant unmet need 4-5 With potential to be the first oral treatment for a range of complement-driven diseases, complement factor B inhibitor iptacopan targets the underlying cause of these conditions through its action on the complement system’s alternative pathway 6,7 ...