FDA Clears Ionis’ Dawnzera as First RNA Preventive for HAE

The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals’ Dawnzera, the first RNA-targeted therapy cleared to prevent attacks of hereditary angioedema in patients aged 12 and older. The once-monthly or every-other-month injectable, known generically as donidalorsen, is the second medicine Ionis will market on its own. In a Phase 3 study, Dawnzera cut monthly attack rates by 81% versus placebo, with similar benefits seen in an open-label extension. The prescribing information carries a warning for anaphylaxis after two cases were recorded in an open-label trial, although the reactions resolved quickly. The approval positions Ionis against established prophylactic treatments sold by Takeda and BioCryst in a market that has seen a string of new entrants this year. Ionis said pricing details will be released later on Thursday.