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Novartis' Iptacopan (LNP023) Receives the US FDA's Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G

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Novartis' Iptacopan (LNP023) Receives the US FDA's Breakthrough Therapy Designation for PNH and Rare Pediatric Disease Designation for C3G

Shots:

  • The US FDA has granted BTD in paroxysmal nocturnal hemoglobinuria and RPDD in C3 glomerulopathy
  • The BTD is based on results from two ongoing P-II studies- where iptacopan showed benefits both in patients who remained anemic and dependent on transfusions despite SOC anti-complement treatment as well as monothx. in anti-C5 naïve PNH patients
  • Iptacopan is a first-in-class- orally administered- potent and highly selective factor B inhibitor of the alternative complement pathway with its first FDA filing anticipated in 2023

 ­ Ref: Novartis | Image: Novatis

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