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
Latest Information Update: 17 Jun 2025
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.