PNH unmet needs

PNH is associated with a significant clinical and economic burden.¹

Left untreated, people living with PNH are at increased risk of life-threatening complications and experience a significant QoL impact with high frequency and severity of daily symptoms.¹

They key sources of burden for PNH patients include:¹

• Risk of thrombosis: A burden of disease study in Europe (n=71) reported that 38% of patients had experienced at least one thrombotic event in their lifetime^{1 2}
• Transfusion risks: The need for blood transfusions is quite common for people with PNH due to the high prevalence of chronic anaemia. Transfusions are associated with a risk of reaction, development of antibodies and iron overload (21% of patients)³ 
• Severe fatigue: The most common symptom in people living with PNH despite current available treatment options.
• Economic impact: Both at an individual patient level, due to an impaired ability to work, and at a healthcare system level, given the significant requirements for medical interventions.
• Risk of thrombosis: A burden of disease study in Europe (n=71) reported that 38% of patients had experienced at least one thrombotic event in their lifetime^{1 2}