Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry

Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the Non-interventional IPIG PNH Registry

This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.

This multinational, non-interventional, descriptive single-arm cohort study is based on secondary analysis of data collected within the iptacopan silo of the IPIG PNH Registry (data on iptacopan-treated patients made available to Novartis). This is a non-interventional study utilizing secondary data and is considered a "registry-based study." The IPIG PNH Registry (CT.gov NCT06524726), the parent registry, includes a dedicated drug silo to collect data from patients using iptacopan in routine care.

Inclusion Criteria:

• Signed informed consent to participate in the IPIG PNH Registry
• PNH confirmed by flow cytometry
• Incident users of iptacopan
• Aged at least 18 years at the iptacopan initiation

Exclusion Criteria:

• Participation in an interventional clinical trial