IPTACOPAN ASH UPDATE

The path to PNH diagnosis and treatment involves many steps and can take months to years Delays in diagnosis and treatment of PNH Up to 3 years to diagnose PNH (avg. 7-9 months) ■ Median age at disease onset 36 years¹ ■ Common symptoms (e.g., fatigue, hemoglobinuria) can have multiple causes ■ "Watch & Wait" for disease progression before treatment is initiated ■ Patients still experiencing symptoms and may be receiving transfusions Increase urgency to intervene earlier Limited options available for treatment ■ Until 2021, only anti-C5's available for treatment ■ 4-6 weeks to determine if treatment is working ■ Some patients unwilling to commit to regular infusions More choice in first line and switch Need to make treatment for life manageable ☐ Regular monitoring with stable patients every 3 months but unstable patients as often as weekly ■ "Good enough" patient outcomes accepted Managed by hematologists Unburden patients from infusions and expect more from treatments 1. 5. Schrezenmeier H et al. Ann Hematol. 2020;99(7):1505-1514. Source: Patient journey market research 2022 26 IPTACOPAN ASH UPDATE | DECEMBER 13, 2022 NOVARTIS | Reimagining Medicine

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