Firazyr (icatibant injection) has been approved by the FDA for acute attacks of hereditary angioedema in adults aged 18+ years. Icatibant is a potent and selective bradykinin B2 receptor antagonist - it inhibits the effects of bradykinin, which is thought to cause HAE (hereditary angioedema) symptoms of inflammation, pain and localized swelling; it treats the clinical symptoms of an acute attack.

Hereditary angioedema, often referred to as HAE, is caused by the improper function or low levels of C1 inhibitor, a protein involved in regulating how certain blood clotting pathways and immune systems function. Fewer than 30,000 individuals in the USA are thought to have HAE. It is a rare, genetic disease which is characterized by recurring episodes of acute swelling which can sometimes be disfiguring and frequently painful - in some cases episodes can be life-threatening.

Swelling typically occurs in the face, extremities, voice box, genitals, windpipe, or gastrointestinal tract, but can affect any part of the body. When the gastrointestinal tract is affected the patient experiences vomiting, nausea and abdominal pain. When there is swelling in the airway the individual can suffocate.

Curtis Rosebraugh, M.D., M.P.H., director of the Office of Drug Evaluation II in the FDA's Center for Drug Evaluation and Research, said:

"Firazyr provides a new option to treat acute attacks of HAE and because it can be self-administered through an injection in the abdominal area, patients can treat themselves upon recognition of an HAE attack."

The FDA reviewed three controlled clinical trials involving 225 patients who were administered 1,076 doses of 30 mg Firazyr. Patients reported onset of symptom relief within two hours of receiving Firazyr, compared to 20 hours for those on placebo.

This is the third drug the FDA has approved for HAE attacks. Berinert was approved in October 2009 for the treatment of abdominal and facial attacks, and Kalbitor, for acute attacks in patients aged 16+ years was approved in December 2009.

Firazyr includes patient counseling information and injection instructions.

Side effects associated with Firazyr include rash, dizziness, increased liver enzymes, infection site reactions, and fever.

Timothy Craig, Professor of Medicine & Pediatrics, Penn State Hershey Medical Center, said:

"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. FIRAZYR is a treatment with demonstrated efficacy that can be carried and stored at room temperature and self-injected by the patient. FIRAZYR addresses this important unmet need by providing HAE patients with fast access to acute treatment."

U.S. HAE Association Executive Vice President Janet Long, said:

"The unpredictability of HAE attacks can place limits on patients' travel, employment opportunities, educational opportunities, and social life. With this approval, patients have an important new option to treat HAE attacks."

Shire Human Genetic Therapies Inc., the marketers of the drug, explained that Firazyr comes in pre-filled syringes that can be stored in temperatures up to 77 Fahrenheit, making it a portable and accessible medication for immediate treatment of HAE attacks.

Sylvie Grégoire, President of Shire Human Genetic Therapies, said:

"Shire has extensive experience meeting the unique needs of patients with rare diseases due to our leadership in this area. With FIRAZYR now approved in 38 countries, we are pleased to bring both this novel treatment and our comprehensive OnePath® patient service and support program to U.S. HAE patients."

Regulatory authorities in 38 countries, including the European Union and now the USA have approved Firazyr for acute HAE attacks in adult patients.

Patients are trained on how to self-administer the Firazyr injection. The majority of patients respond to one dose. Sometimes, up to two additional doses may be administered within a twenty-four hour period. If this is necessary, each additional dose must be done at least six hours apart.

Sources: Shire, FDA.

View drug information on Berinert; Kalbitor.