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A Multicenter, Single Arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Have Hb≥10 g/dL in Response to Anti-C5 Antibody and Switch to Iptacopan

Trial Profile

A Multicenter, Single Arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Have Hb≥10 g/dL in Response to Anti-C5 Antibody and Switch to Iptacopan

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 17 Jun 2025

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At a glance

  • Drugs Iptacopan (Primary)
  • Indications Paroxysmal nocturnal haemoglobinuria
  • Focus Registrational; Therapeutic Use
  • Acronyms APPULSE; APPULSE-PNH
  • Sponsors Novartis Pharmaceuticals

Most Recent Events

  • 12 Jun 2025 According to a Novartis media release, company will present positive result data from this study at the European Hematology Association (EHA) Congress 2025.
  • 12 Jun 2025 Results presented in the Novartis media release.
  • 06 Dec 2024 According to a Novartis media release, Findings from APPULSE-PNH build on the Phase III program showing the efficacy and safety of Iptacopan in adults with PNH1, which is approved in the US, EU, Japan and China.

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