The FDA has approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE).

Hereditary angioedema, which is caused by having insufficient amounts of a plasma protein called C1-esterase inhibitor, affects approximately 6,000 to 10,000 people in the U.S. People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract or airway. These acute attacks of swelling can occur spontaneously, or can be triggered by stress, surgery or infection. Swelling of the airway is potentially fatal without immediate treatment.

Ruconest is a human recombinant C1-esterase inhibitor purified from the milk of genetically modified (transgenic) rabbits. Ruconest is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling.  

The safety and efficacy of Ruconest was evaluated in a multicenter controlled clinical trial. Forty-four adult and adolescent patients with acute attacks were treated with Ruconest. The most common adverse reactions reported in patients treated with Ruconest were headache, nausea and diarrhea.

Ruconest received Orphan Drug Designation for acute attacks by the FDA because it is intended for treatment of a rare disease or condition.

Ruconest is manufactured by Netherlands-based Pharming Group, and will be distributed in the U. S. by Santarus, a wholly owned subsidiary of Salix Pharmaceuticals.