FDA Approves Intravenous Therapy for Hereditary Angioedema

Yael Waknine

October 12, 2009

October 12, 2009 — The US Food and Drug Administration (FDA) has approved a plasma-derived C1-esterase inhibitor (C1-INH) concentrate for intravenous injection (Berinert, CSL Behring, Inc) to treat adults and adolescents with acute abdominal attacks and facial swelling associated with hereditary angioedema (HAE).

HAE, a genetic disorder caused by an inherited deficiency of C1-INH, is characterized by relapsing, self-limiting episodes of edema that occur primarily in subcutaneous tissue, the intestinal wall, and larynx. Abdominal symptoms include severe pain, nausea, vomiting, cramps, and diarrhea; attacks that involve the face and throat can result in airway obstruction, asphyxiation, and death if left untreated.

"For individuals with HAE, episodes of swelling can be extremely painful and frightening," said Timothy Craig, MD, professor of medicine and pediatrics, Penn State Hershey Medical Center, Hershey, Pennsylvania, in a company news release. "With the approval of Berinert, healthcare professionals can now provide HAE patients in the US with a safe and effective treatment option that rapidly relieves the symptoms of acute attacks in the face and abdomen."

FDA approval was based on data from the phase 2/3, double-blind, placebo-controlled International Multi-center Prospective Angioedema C1-Inhibitor Trial (IMPACT) study of 124 patients showing that treatment with C1-INH significantly decreased the median time to symptom relief relative to placebo (30 minutes vs 1.5 hours).

Adverse events most commonly reported in the study occurred in 4% to 7% of treated patients and included subsequent HAE attack, headache, abdominal pain, nausea, muscle spasms, pain, diarrhea, and vomiting. The most serious adverse reaction was an increase in HAE pain severity.

Because of the risk for hypersensitivity reactions, epinephrine should be readily available for use if C1-INH administration is discontinued for symptoms such as generalized urticaria, tightness of the chest, wheezing, hypotension, and/or anaphylaxis.

The FDA warns that thrombotic events have occurred in patients receiving off-label high doses of C1-INH. Patients with known risk factors for these events should be carefully monitored.

As with other products made from human plasma, the risk for transmission of viruses and, theoretically, the Creutzfeldt-Jakob disease agent cannot be completely eliminated.

Marketed as Berinert P, C1-INH injection previously was approved for use in Germany, Austria, Switzerland, and several other countries.

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