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A recombinant C1-esterase inhibitor to be sold as Ruconest has expanded the list of therapeutic options for hereditary angioedema (HAE), following FDA approval.

Ruconest won approval as treatment of acute symptomatic episodes of HAE in adults and adolescents. A potentially life-threatening condition, HAE affects an estimated 6,000 to 10,000 Americans. Genetic defects that prevent normal production of endogenous C1-esterase inhibitor are responsible for the condition.

HAE symptoms may arise spontaneously or in response to stress, infection, or other factors. An episode can evolve rapidly, leading to severe inflammation-driven edema affecting the hands, extremities, face, gastrointestinal tract, and larynx.

According to statements from the FDA and U.S. distributor Salix Pharmaceuticals, data submitted in support of the approval application included results of a randomized clinical trial involving 44 patients with a combined total of 170 acute HAE episodes. Ruconest-treated patients had a median time to onset of symptom relief of 90 minutes as opposed to 152 minutes in placebo-treated patients (P=0.031).

Derived from the milk of transgenic rabbits, Ruconest counters HAE symptoms by restoring plasma C1-esterase inhibitor to normal levels.

Principal adverse effects associated with the C1-esterase inhibitor were headache, nausea, and diarrhea.

Recombinant C1-esterase inhibitor is manufactured by Pharming Group in Leiden, the Netherlands, and licensed to Santarus, a Salix subsidiary.

Ruconest joins the human plasma-derived C1 inhibitors Cinryze and Berinert, the kallikrein inhibitor ecallantide (Kalbitor), and the bradykinin receptor antagonist icatibant (Firazyr) as approved therapies for HAE.

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