Hereditary angioedema, the drug that affects RNA

Hereditary angioedema, the drug that affects RNA


A rare and dangerous genetic disease, which affects from childhood or early adolescence. We are talking about hereditary angioedema, in which edema suddenly appears on the skin and in internal organs – such as the stomach and intestines, esophagus -, in the face and throat, and which can even lead to death. This is why it is important to develop new drugs capable of counteracting it. On this front, good results come from ongoing studies on donidalorsen, an innovative experimental drug.

The new results

As reported by the companies Otsuka Pharmaceutical Italy and Ionis, the drug achieved the objective of statistically significantly reducing the rate of angioedema attacks in patients treated with donidalorsen (administered by subcutaneous injection every 4 or 8 weeks) compared to placebo. The phase 3 OASIS-HAE study also showed a favorable safety and tolerability profile, and no serious adverse events occurred in patients treated with donidalorsen. Also ongoing is the OASIS-Plus study, which includes the open-label extension of the Phase 3 study, and a separate cohort of patients who switched to donidalorsen from other therapies for attack prophylaxis (switching cohort). The results of both studies are expected to be presented in detail around the middle of the year.

What is donidalorsen

Donidalorsen is an experimental drug whose target is RNA, designed as a prophylactic (i.e. preventative) therapy to precisely target and silence the production of the protein prekallikrein (PKK), which plays an important role in the activation of inflammatory mediators associated with acute attacks. In this way, the drug is able to interrupt the path that leads to the sudden formation of edema. Donidalorsen has received orphan drug designation in the United States, while such evaluation is ongoing in the European Union.

The approval process in the USA and Europe

Otsuka holds the exclusive rights for the marketing of donidalorsen in Europe, and is preparing the documents to request authorization from the European Agency (EMA), while Ionis, which is conducting the studies, is preparing the request for New Drug Application to US Food and Drug Administration (FDA). “Despite the availability of other prophylactic treatments, a substantial number of patients with hereditary angioedema continue to experience unpredictable, painful and severe attacks,” says Andy Hodge, CEO, Otsuka Pharmaceutical Europe. “We believe that donidalorsen, if approved, could address this unmet medical need and provide a potential new therapeutic option for these patients.”



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