Positive review for Fabhalta

The European Medicines Agency has issued a positive opinion for Fabhalta (iptacopan), a monotherapy for a rare genetic blood disorder which has shown superior activity to the standard of care. The disorder is paroxysmal nocturnal haemoglobinuria (PNH) which causes the premature breakdown of red blood cells in the body leading to fatigue, shortness of breath and often requiring blood cell transfusions.