Novartis investigational oral therapy iptacopan (LNP023) receives FDA Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G

Novartis announced that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G).

The breakthrough designation is intended to expedite the development and review of medicines for serious conditions to address unmet medical need, where early clinical evidence indicates a drug may demonstrate substantial improvement over available therapy on clinically significant endpoints. The FDA granted BTD to iptacopan for the treatment of PNH based on positive interim results from two ongoing Phase II studies, where iptacopan showed substantial benefits both in patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment, as well as monotherapy in anti-C5 naïve PNH patients.

PNH is a rare and life threatening blood disorder characterized by complement-driven hemolysis, thrombosis and impaired bone marrow function, resulting in anemia, fatigue and other debilitating symptoms that can impact patients’ quality of life. Despite current standard of care – anti-C5 therapy eculizumab or ravulizumab – a large proportion of PNH patients remain anemic and dependent on transfusions.

FDA grants the rare pediatric designation for serious or life-threatening diseases primarily affecting individuals aged 18 years or younger and impacting fewer than 200,000 people. C3G, an ultra-rare and severe form of primary glomerulonephritis, is characterized by complement dysregulation. It has a poor prognosis; about 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience disease recurrence post kidney transplant. It has a worldwide annual incidence of 1-2 per million.