- The CHMP’s positive opinion is based on a P-III study assessing Ultomiris and showed effectiveness in completing C5 complement inhibition @26wks. for children & adolescents aged ≤18yrs. with PNH
- The study demonstrated that there is no reported treatment-related sAEs, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis
- The CHMP recommended the expanded use of Ultomiris to include children (with a body weight of ≥10kg) and adolescents with PNH who experience hemolysis with clinical symptoms indicative of high disease activity and for individuals who are clinically stable after being treated with Soliris for the past 6mos.
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