AZN - EMA advisory committee backs Alexion's Ultomiris for blood disorder in children

Lisa Maree Williams/Getty Images News AstraZeneca (AZN) announces that Alexion’s (ALXN) Ultomiris (ravulizumab) has been recommended for marketing authorization in the European Union ((EU)) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria ((PNH)). The EMA's Committee for Medicinal Products for Human Use ((CHMP)) based its positive opinion on interim results from Phase III trial in children and adolescents with PNH. The trial demonstrated that Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks. The EMA endorsement is to include children with a body weight of 10 kg or above, adolescents who experience haemolysis with clinical symptoms indicative of high disease activity and those individuals who are clinically stable after having been treated with another Alexion drug, Soliris, for at least the past six months. PNH is an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause

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EMA advisory committee backs Alexion’s Ultomiris for blood disorder in children