FDA Approves New Rx for Rare Genetic Disorder

Ruconest approved to treat acute attacks of hereditary angioedema in adults and teenagers

/ Author:  / Reviewed by: Joseph V. Madia, MD Beth Bolt, RPh

(RxWiki News) The US Food and Drug Administration (FDA) has approved a new medication to treat a rare but serious genetic disorder.

The FDA announced that it has approved Ruconest for the treatment of acute attacks of hereditary angioedema (HAE) in adults and teens.

HAE is a rare but potentially dangerous disorder passed down through families. The condition causes repeated attacks of severe swelling, often in the face and airways. While minor trauma or stress can trigger one of these attacks, the swelling often happens without any known trigger.

Until recently, there were limited options for treating HAE.

"Ask a pharmacist about new treatments for your disease."

“Hereditary angioedema is a rare and potentially life-threatening disease,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research, in a press statement. “Today’s approval provides an important treatment option for these patients.”

HAE occurs due to low levels of a plasma protein called C1-esterase inhibitor.

Ruconest is the first recombinant C1-esterase inhibitor product approved to treat attacks of HAE.

The FDA approval of Ruconest comes after a clinical trial of the medication's safety and effectiveness in 44 adults and teens with acute attacks. The most common side effects seen in patients treated with Ruconest were headache, nausea and diarrhea.

Ruconest is manufactured by Pharming Group NV in the Netherlands. The medication will be distributed in the US by Santarus, Inc., which is owned by Salix Pharmaceuticals, Ltd.

Review Date: 
July 17, 2014
Last Updated:
July 18, 2014