Overview

• The Italian Medicines Agency has greenlit reimbursement for iptacopan in adult PNH patients who remain anemic after at least three months on C5 inhibitors.
• Results from the phase 3 Apply-PNH trial demonstrated significant hemoglobin gains and a marked reduction in red-cell transfusions versus continued C5 inhibitor therapy.
• Iptacopan inhibits Factor B of the alternative complement pathway to address extravascular hemolysis that C5 blockers leave untreated.
• Oral once-daily dosing frees patients from hospital infusions and supports greater autonomy, daily functioning and quality of life.
• Novartis will collaborate with regional health authorities to ensure timely and equitable patient access to the new treatment across Italy.