Donidalorsen for Hereditary Angioedema

This trial tests the safety and effectiveness of donidalorsen for individuals with hereditary angioedema (HAE), a condition causing sudden swelling attacks. Researchers aim to determine if varying doses or dosing schedules are more effective over time. Participants must have completed a previous related study and should have access to emergency medication for HAE attacks. Those who have successfully finished the earlier study and have HAE might be suitable for this trial. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

The trial information does not specify if you need to stop taking your current medications. However, you must have access to at least one acute medication to treat angioedema attacks, and any changes in your medication that could affect your participation might make you unsuitable for the study.

Research has shown that donidalorsen was well-tolerated in earlier studies. In one study, donidalorsen successfully lowered certain blood levels without raising safety concerns. Another study with 171 participants found that donidalorsen reduced swelling attacks in people with hereditary angioedema compared to a placebo, with no major safety issues reported.

Unlike the standard treatments for hereditary angioedema, which generally focus on managing symptoms or preventing attacks through C1 inhibitor replacement or kallikrein inhibition, Donidalorsen offers a unique approach. Researchers are excited because Donidalorsen targets and reduces the production of prekallikrein, a key protein involved in the pathway that causes swelling in hereditary angioedema. This mechanism of action not only promises more effective control of symptoms but could also offer a longer-lasting solution with less frequent dosing. Its administration via subcutaneous injection for extended periods could make managing the condition more convenient and potentially improve the quality of life for patients.

Research has shown that donidalorsen, the treatment under study in this trial, reduces the number of swelling attacks in people with hereditary angioedema (HAE). One study found that patients taking donidalorsen had fewer attacks than those taking a placebo, a harmless pill with no active medicine. Another study revealed that patients who switched to donidalorsen from other treatments experienced a 62% further drop in their monthly attack rates. Over two years, donidalorsen was well tolerated, with no new safety issues reported. These findings suggest that donidalorsen could be a promising option for managing HAE.¹⁴⁶⁷⁸