The US Food and Drug Administration has approved the first monoclonal antibody (mAb) to prevent hereditary angioedema (HEA) attacks. Dublin-based Shire was given the go-ahead in August to market Takhzyro (lanadelumab) as prophylactic treatment for HEA types I and II in patients aged 12 years or older. HEA is a rare but life-threatening genetic disease caused by mutations in the C1 esterase inhibitor gene, which leads to an overactivation of the complement system. This can trigger unpredictable bouts of subcutaneous or submucosal swelling anywhere in the body, potentially leading to blocked airways, and in the gut the attacks can cause intense pain, vomiting and dehydration.

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Fonte: NATURE BIOTECHNOLOGY VOLUME 36 NUMBER 11 NOVEMBER 2018