Donidalorsen, formerly known as IONIS-PKK-LRx, is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to target the production of prekallikrein (PKK), which plays an important role in the activation of inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). By reducing the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks.
About Hereditary Angioedema (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 20,000 people in the U.S. and Europe.
Safety and efficacy have not been evaluated by any regulatory authorities for the indication described.