Donidalorsen, formerly known as IONIS-PKK-LRx, is an investigational ligand-conjugated antisense (LICA) medicine designed to target the prekallikrein (PKK) pathway.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disease that is characterized by severe and potentially fatal swelling of the arms, legs, face and throat. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of HAE. By inhibiting the production of PKK, donidalorsen could be an effective prophylactic approach to preventing HAE attacks. 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 indications described.