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RELEASE
Donidalorsen (Dawnzera – Ionis), a subcutaneously injected prekallikrein-directed antisense oligonucleotide, has been approved by the FDA for prevention of hereditary angioedema (HAE) attacks in patients ≥12 years old. It is the first RNA-targeted drug to be approved in the US for this indication.
THE DISEASE ― HAE, an autosomal dominant disorder with an estimated prevalence of 1:50,000, is characterized by recurrent, often unpredictable, painful attacks of angioedema, typically involving the skin and the gastrointestinal, oropharyngeal, and laryngeal mucosa. Attacks generally last for 2-5 days and are less likely to be fatal now that effective treatment is available.
Uncontrolled production of the potent vasodilator bradykinin has been shown to be the mediator of angioedema attacks. HAE is usually caused by a mutation of the SERPING 1 gene, which results in deficiency …







