December 21, 2020
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FDA grants breakthrough therapy status to LNP023 for paroxysmal nocturnal hemoglobinuria

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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.