Hereditary Angioedema Market Snapshot: Room For Pills And Injections As Prophylaxis Makes Gains
While many HAE patients converted from injectable prophylaxis to BioCryst’s oral Orladeyo last year, physicians said a promising injectable candidate from Ionis could benefit from varied patient preference.
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The US firm has sold some of its royalty stream for the gene therapy Spinraza and cardiovascular drug candidate, pelacarsen, to Royalty Pharma for $500m upfront, which should help fund upcoming milestones for Ionis’ late-stage pipeline.
Based on top-line Phase III data for garadacimab, CSL is preparing to submit the Factor XIIa inhibitor for US FDA approval for monthly prophylaxis of hereditary angioedema and expand the firm’s HAE franchise.
The company announced data from the Phase III SPRING study of Takhzyro in children aged 2-11; there is no approved long-term prophylactic therapy for children under 6.