First oral treatment against residual haemolytic anaemia in patients with paroxysmal nocturnal haemoglobinuria

1. März 2024 – EMA has recommended granting a marketing authorisation in the European Union (EU) for Voydeya, the first oral treatment for patients with paroxysmal nocturnal haemoglobinuria (PNH) who continue to have haemolytic anaemia (low levels of red blood cells) despite treatment with a C5 complement inhibitor (eculizumab or ravulizumab).

PNH is a rare genetic disorder and potentially life-threatening blood disease leading to the premature destruction of red blood cells (haemolytic anaemia) by the immune system. Disease symptoms include fatigue, body pain, blood clots, bleeding and shortness of breath. PNH generally worsens over time and patients often require red blood cell transfusions. The standard treatment for PNH is monoclonal antibodies (ravulizumab or eculizumab) known as complement inhibitors. Some patients who experience residual haemolytic anaemia need subcutaneous infusions of another complement inhibitor (pegcetacoplan) to prevent the damage to red blood cells.

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