FDA approves Ruconest to treat patients with hereditary angioedema

The FDA has approved the first recombinant C1-esterase inhibitor for treating acute attacks of swelling in adults and adolescents with hereditary angioedema, according to a press release.

“Hereditary angioedema (HAE) is a rare and potentially life-threatening disease,” Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, said in the release. “[The] approval provides an important treatment option for these patients.”

Karen Midthun

C1-esterase inhibitor (Ruconest, Salix Pharmaceuticals/Pharming Group NV) is an injectable human recombinant purified from genetically modified rabbits’ milk, according to the release. It is indicated to restore a functional level of C1-esterase inhibitor in a patient’s plasma and treat acute attacks of swelling. It was approved at 50 IU/kg dosage.

HAE is caused by insufficient C1-esterase inhibitor plasma protein and affects approximately 6,000 to 10,000 people in the US, the release said. Symptoms include rapid swelling of the hands, feet, limbs, face, intestinal tract or airway, with acute attacks occurring spontaneously or through stress, surgery or infection.

In a multicenter controlled clinical trial, 44 adults and adolescents with acute HAE were treated with Ruconest to evaluate its safety and efficacy. Common adverse reactions were headache, nausea and diarrhea.

“Results … demonstrate Ruconest is a safe and effective option for the treatment of acute hereditary angioedema attacks,” researcher Marc Riedl, MD, of the US HAEA Angioedema Center, University of California, San Diego, said in a separate press release. “We strive to better the lives of those suffering from hereditary angioedema and part of that is ensuring patients have access to advanced treatments that have been proven to work in clinical trials. Ruconest is an important addition to those treatment options.”