PNH is a rare, chronic, life-threatening disease affecting the complement cascade – a critical part of the normal innate immune response.⁷ 

Uncontrolled terminal complement activation impairs the normal functioning of blood cells and leads to life-threatening consequences such as IVH, thromboembolic events, renal impairment and pulmonary hypertension.^6,8-10

In IVH, red blood cells are destroyed within the bloodstream, releasing haemoglobin into plasma and causing life-threatening consequences such as thrombosis and organ damage.^13–15 Elevated LDH level (≥1.5 x ULN) is a key biomarker for IVH.^13-15 

If PNH is left untreated, it can lead to early mortality.^4,16 Thrombosis is a leading cause of death, occurring in 40-67% of patients, and can be fatal at the first event.^†17,18

Before the availability of today’s standard of care of C5 inhibitors, ~35% of patients died within 5 years.^‡16,19

^*Complete C5 inhibition is defined as serum free C5 concentration <0.5 μg/mL.^2,3

^†Percentages listed are from various studies, and many do not account for racial variance.^17,18

^‡Based on the natural history study of 80 patients with PNH in London, UK, between 1940–1970 who were followed for up to 48 years after diagnosis.¹⁹