FDA approves Ionis’ Dawnzera as first RNA-targeted therapy for HAE

The US Food and Drug Administration (FDA) has approved Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older.

Developed by California-based drugmaker Ionis (Nasdaq: IONS), Dawnzera is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE. Dawnzera 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W).

HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the USA.