FDA grants breakthrough therapy status to LNP023 for paroxysmal nocturnal hemoglobinuria
Click Here to Manage Email Alerts
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.
Read more about
Click Here to Manage Email Alerts