FDA Approves Ruconest for Hereditary Angioedema
July 17, 2014 -- The U.S. Food and Drug Administration yesterday 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 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.
“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.”
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 Pharming Group NV, Leiden, the Netherlands, and will be distributed in the United States by Santarus Inc., a wholly owned subsidiary of Salix Pharmaceuticals Inc., Raleigh, North Carolina.
Source: FDA
Posted: July 2014
Related articles
- Santarus and Pharming Announce Receipt of FDA Refusal to File Letter for Rhucin Biologics License Application - February 28, 2011
- Pharming And Santarus Announce Submission Of Rhucin Biologics License Application to FDA - December 28, 2010
- Pharming Provides Update on Rhucin - January 29, 2008
Ruconest (C1 esterase inhibitor (recombinant)) FDA Approval History
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.
