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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 Are Naive to Complement Inhibitor Therapy

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Trial Profile

A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

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 APPOINT-PNH
  • Sponsors Novartis Pharma A.G.; Novartis Pharmaceuticals

Most Recent Events

  • 12 Jun 2025 According to a Novartis media release, company will present data from this study at the European Hematology Association (EHA) Congress 2025.
  • 14 Apr 2025 According to a Novartis media release, based on data from this and APPLY-PNH studies Health Canada approved Fabhalta (iptacopan capsules), first oral treatment for adult patients with PNH
  • 13 Feb 2025 According to a Novartis media release, company nce that Health Canada has granted a Notice of Compliance for Fabhalta (iptacopan capsules) as the first oral monotherapy treatment for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia, based on the Phase III APPLY-PNH trial and supported by the Phase III APPOINT-PNH study.

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