FDA approves icatibant to treat acute attacks of hereditary angioedema

FDA has approved icatibant (Firazyr, Shire Human Genetic Therapies) injection for the treatment of acute attacks of hereditary angioedema (HAE) in patients 18 years of age and older.

HAE is caused by low levels or improper function of the C1 inhibitor protein, which is involved in regulating how certain immune system and blood-clotting pathways function. There is usually a family history of the condition. Fewer than 30,000 people in the United States have HAE.

HAE is a rare and debilitating genetic disease characterized by recurrent, sometimes disfiguring, and often painful episodes of acute swelling that in some cases can be life-threatening. The swelling attacks can affect any part of the body but most commonly occur on the face, gastrointestinal tract, extremities, or genitals. Laryngeal attacks can be fatal due to risk of suffocation.

“Until now, HAE patients faced challenges gaining rapid access to acute treatment, such as the need to travel to the physician’s office or hospital,” said Timothy Craig, professor of medicine and pediatrics, Penn State Hershey Medical Center, in a Shire press release. “Icatibant is a treatment with demonstrated efficacy that can be carried and stored at room temperature and self-injected by the patient. Icatibant addresses this important unmet need by providing HAE patients with fast access to acute treatment.”

The safety and efficacy of icatibant was demonstrated in 3 controlled clinical trials, with open-label extension periods, in which 225 patients received 1,076 doses of 30-mg icatibant. The median time for patients treated with icatibant to report onset of symptom relief was 2 hours compared to almost 20 hours with placebo. Icatibant is the third drug approved in the United States to treat HAE attacks. In October 2009 FDA approved C1 esterase inhibitor, human (Berinert, CSL Behring GmbH) to treat facial and abdominal attacks of HAE, and ecallantide (Kalbitor, Dyax) was approved in December 2009 to treat acute attacks of HAE in patients aged 16 years and older.

FDA approved icatibant with patient counseling information that includes injection instructions. The most common side effects reported by those using icatibant were injection-site reactions, fever, increased liver enzymes, dizziness, and rash.