FDA Approves Orphan Drug for Rare, Life-threatening Hereditary Angioedema

The US Food and Drug Administration (FDA) recently approved a new drug aimed to aid the thousands of Americans affected by hereditary angioedema (HAE).

The C1 esterase inhibitor, known as Ruconest, is the first medication of its kind approved to help the up to 10,000 people currently diagnosed with HAE and experiencing symptoms that can include swelling of the hands, feet, limbs, face, intestinal tract, or airway. According to a statement from the FDA, these instances can be random or triggered by stress, surgery, or infection and can be fatal when they affect the airway.

Derived from a human recombinant C1 esterase inhibitor that is purified from the milk of genetically modified rabbits, Ruconest is meant to restore functional levels of the inhibitor to a HAE patient’s plasma, which would help during an acute attack of the 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. “Today’s approval provides an important treatment option for these patients.”

Out of a group of 44 adult and adolescent patients treated during a multicenter controlled clinical trial, the most common adverse reactions reported were headaches, nausea, and diarrhea.

Ruconest, which was given an orphan drug designation by the FDA because it treats a rare disease, is manufactured by the Pharming Group NV, in the Netherlands and will be distributed in the United States by Santarus, Inc., a subsidiary of Salix Pharmaceuticals.