Salix Pharmaceuticals Gets FDA Approval for Orphan Drug Ruconest
Salix Pharmaceuticals, Ltd., based in Raleigh, North Carolina, and Pharming Group NV, based in Leiden, the Netherlands, report that US the Food and Drug Administration (FDA) has approved Ruconest (conestat alfa), a recombinant human C1 esterase inhibitor, for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE).
Ruconest is a human recombinant C1-esterase inhibitor purified from the milk of genetically modified (transgenic) rabbits. Ruconest is manufactured by Pharming Group NV. Salix has licensed exclusive rights from Pharming to commercialize the drug in North America and market it for the treatment of acute HAE attack symptoms. Ruconest will be distributed in the United States by Santarus Inc., a wholly owned subsidiary of Salix Pharmaceuticals Inc. Salix currently plans on making Ruconest accessible to patients later in 2014.
Ruconest was granted orphan-drug status by the FDA. HAE, 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 United States. 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.