THURSDAY, July 26, 2018 — A small-molecule inhibitor of plasma kallikrein, BCX7353, results in a significantly lower rate of hereditary angioedema attacks compared with placebo, according to a study published in the July 26 issue of the New England Journal of Medicine.

Emel Aygören-Pürsün, M.D., from University Hospital Frankfurt in Germany, and colleagues randomized 77 patients with type I or II hereditary angioedema with a history of at least two angioedema attacks per month to BCX7353 (at four doses: 62.5, 125, 250, and 350 mg) or placebo. Seventy-two of the patients completed the trial.

The researchers found that patients who received BCX7353 at daily doses of 125 mg or more had a significantly lower rate of confirmed angioedema attacks compared with those who received placebo, with a 73.8 percent difference at 125 mg. The 125- and 250-mg groups had significant benefits with respect to quality-of-life scores. The most commonly reported adverse events were gastrointestinal adverse events, mainly grade 1, especially in the two highest BCX7353-dose groups.

“Once-daily oral administration of BCX7353 at a dose of 125 mg or more resulted in a significantly lower rate of attacks of hereditary angioedema than placebo,” the authors write. “Longer studies will need to be performed to assess the safety profile of long-term dosing.”

The study was funded by BioCryst Pharmaceuticals, the manufacturer of BCX7353.

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Posted: July 2018

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