Donidalorsen, formerly known as IONIS-PKK-LRx, is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of prekallikrein (PKK). PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of hereditary angioedema (HAE). By inhibiting the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks.
About Hereditary Angioedema (HAE)
HAE is a rare and life-threatening genetic disease characterized by unpredictable and frequently severe swelling of the skin, gastrointestinal (GI) tract, upper respiratory system, face, and throat. It is estimated that there are more than 20,000 patients with HAE in the U.S. and Europe.
Safety and efficacy have not been evaluated by any regulatory authorities for the indication described.