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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 Apr 2024

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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

    • 22 Mar 2024 According to an European Medicines Agency media release, EMA has recommended granting a marketing authorisation in the European Union (EU) for Fabhalta (iptacopan), EMA's recommendation is based on the results of two phase III trials (APPOINT-PNH and APPLY-PNH).
    • 22 Mar 2024 According to a Novartis media release, following the CHMP's recommendation to approve Fabhalta in adult patients with PNH who have hemolytic anemia, the European Commission (EC) will take a final decision within approximately two months.
    • 22 Mar 2024 According to a Novartis media release, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting a marketing authorization for Fabhalta (iptacopan) for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia based on robust data from the Phase III APPLY-PNH study and APPOINT-PNH study.

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