Ionis Pharma Wins FDA Nod For First RNA-Targeted Therapy For Rare Genetic Swelling Disorder

On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older.

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.

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 U.S.

Also Read: Ionis Posts Surprise Q2 Profit, Double Revenues On Stronger Sales From Newly Approved Genetic Disease Drug

Dawnzera 80mg is self-administered ...