Ionis Rare Disease Drug Is Headed to FDA, But Forthcoming Data Will Decide Competitive Profile

Ionis Pharmaceuticals has reported positive results from the Phase 3 trial of its drug candidate, donidalorsen, for the treatment of hereditary angioedema (HAE). The drug met its main trial goal of reducing the rate of HAE attacks, was well tolerated by patients, and no serious adverse events were reported in the treatment group. Phase 3 results will be presented at an upcoming medical meeting. The drug is designed to target the mRNA responsible for producing a protein called prekallikrein, a precursor to the kallikrein protein that contributes to HAE attacks. If approved, donidalorsen would compete with other HAE drugs on the market. The commercialization of the drug will be handled by Ionis in the U.S. and by Otsuka Pharmaceutical in Europe.

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