FDA grants breakthrough therapy status to LNP023 for paroxysmal nocturnal hemoglobinuria
The FDA granted breakthrough therapy designation to iptacopan for treatment of paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare but life-threatening blood disorder associated with complement-driven hemolysis, thrombosis and poor bone marrow function. This often leads to anemia, fatigue and other symptoms that can reduce patients’ quality of life.
Standard treatment consists of anti-C5 therapies eculizumab (Soliris, Alexion) or ravulizumab (Ultomiris, Alexion). However, even with these therapies, a considerable percentage of patients remain anemic and transfusion dependent.
Iptacopan (LNP023, Novartis) is a first-in-class highly selective factor B inhibitor of the alternative complement pathway.
The FDA based the breakthrough therapy designation for iptacopan on positive interim results of two ongoing phase 2 studies.
The studies showed iptacopan conferred substantial benefit for patients with PNH who remained anemic and dependent on transfusions despite standard anti-complement treatments. In addition, the agent showed effectiveness as monotherapy for patients with anti-C5-naive patients.
The FDA also granted rare pediatric disease designation to iptacopan for treatment of C3 glomerulopathy.
C3G is a rare form of primary glomerulonephritis associated with complement dysregulation.
Approximately half of patients progress to end-stage renal disease within 10 years, and up to 70% of patients develop recurrence after kidney transplant.
The FDA grants rare pediatric designation to agents intended to treat serious or life-threatening diseases that affect fewer than 200,000 people, many of whom are aged 18 years or younger.