FDA Panel Approves New Drug Ruconest to Treat Rare Genetic Disease

The U.S. Food and Drug Administration panel members have approved a new drug, Ruconest, which treats rare genetic disease called hereditary angioedema.

Ruconest, the human recombinant C1-esterase inhibitor, is the first to be approved by the FDS for the treatment of acute attacks in adults and adolescent patients with hereditary angioedema (HAE). Ruconest is purified from the milk of genetically-modified rabbits. This novel product is designed to restore the level of functional C-1 esterase inhibitor in a patient's plasma, eventually treating the acute attack of swelling.

Hereditary angioedema (HAE) occurs due to insufficient amount of a plasma protein called C1-esterase inhibitor that affects approximately 6,000-10,000 people in the United States.  People with HAE are known to develop rapid swelling in the hands, feet, limbs, face, intestinal tract or airway. These acute attacks occur spontaneously or are triggered by stress, surgery or infection.  But swelling of the airway is known to be potentially fatal without immediate treatment.

"Hereditary angioedema is a rare and potentially life-threatening disease," said Karen Midthun, M.D., director of the FDA's Center for Biologics Evaluation and Research. "Today's approval provides an important treatment option for these patients."

The authorities evaluated the safety and efficacy of the new product using a controlled clinical trial. The trail included 44 adults and adolescent patients with acute attacks who were treated with Ruconest. The common adverse reaction reported by the participants included headache, nausea and diarrhea.

Since the drug is intended to treat a rare disease or condition, it received an orphan drug designation for acute attacks by the FDA.

Ruconest is manufactured by Pharming Group NV and will be distributed in the U.S. by Santarus Inc.