First oral monotherapy for patients with paroxysmal nocturnal haemoglobinuria

29 mars 2024 – EMA has proposed granting marketing authorization within the European Union (EU) for Fabhalta (iptacopan), an oral medication designed for adults diagnosed with paroxysmal nocturnal hemoglobinuria (PNH) who are experiencing hemolytic anemia.

PNH, a rare genetic condition, triggers the premature destruction of red blood cells (hemolytic anemia) by the immune system, posing potential life-threatening risks. Symptoms of the disease encompass fatigue, bodily discomfort, blood clotting, hemorrhaging, and respiratory difficulties. Typically, PNH progresses over time, often necessitating red blood cell transfusions for patients.

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