At the 22nd Congress of the European Hematology Association (EHA), researchers presented new data from the International Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry.
Data showed real-world evidence of benefits of treatment with rare diseases specialist Alexion Pharmaceuticals’ (Nasdaq: ALXN) Soliris (eculizumab), a complement inhibitor, for patients with PNH regardless of high disease activity (HDA), and for patients with hemolytic PNH and those with PNH with concurrent aplastic anemia (AA). A consensus-based algorithm developed by international PNH experts for the diagnosis of PNH was also presented.
PNH is an ultra-rare, auto-immune blood disorder that strikes people of all ages, with an average age of onset in the early 30s, and is characterized by complement-mediated hemolysis (destruction of red blood cells). Net product sales of Soliris were up 9.7% to $2.84 billion, for full-year 2016.
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