The FDA ( Food and Drug Administration ) has approved a label expansion for self-administration of Berinert, C1 Esterase Inhibitor ( Human ), a pasteurized, nanofiltered therapy indicated for the treatment of acute attacks of hereditary angioedema ( HAE ), a rare and potentially fatal genetic disorder. With appropriate training from a physician, patients can now self-administer Berinert by intravenous infusion. As part of the label expansion, Berinert is now also indicated to treat life-threatening laryngeal HAE attacks, as well as facial and abdominal attacks.

Intervention with Berinert at the onset of an abdominal or facial HAE attack has been shown to provide significantly faster relief to a patient ( median onset of relief of 48 minutes in clinical trials, versus more than four hours in placebo group ). In a non-placebo extension study, the use of Berinert to treat laryngeal attacks provided a median onset of relief of 15 minutes, which is significant due to the potentially life-threatening nature of these attacks.

CSL Behring has marketed Berinert in the United States for the treatment of acute abdominal or facial attacks of HAE in adolescent and adult patients since its FDA approval in October 2009. Approval of the expanded label for Berinert follows years of clinical experience in Europe establishing the safety and efficacy of self-administration of C1-INH therapy.

Berinert, C1 Esterase Inhibitor ( Human ), a pasteurized, nanofiltered, plasma-derived intravenous therapy, treats acute facial, abdominal and laryngeal hereditary angioedema symptoms by providing C1-INH deficient adult and adolescent patients with the missing human protein. Without C1-INH, people with hereditary angioedema suffer from recurrent episodes of rapid swelling of areas of the skin and underlying tissues including the face, mouth, throat and abdomen.
CSL Behring has marketed its pasteurized and nanofiltered C1-esterase inhibitor concentrate, Berinert, in Germany for more than 30 years. The product is also approved and marketed in 23 other European countries, the United States, Australia, Canada, Israel, Argentina, Japan and South Korea.

The safety and efficacy of Berinert for prophylactic therapy have not been established. Berinert is contraindicated in individuals with a history of life-threatening systemic reactions, to C1 esterase inhibitor preparations, including anaphylaxis.
The most serious adverse reaction reported in subjects who received Berinert in clinical studies was an increase in the severity of pain associated with hereditary angioedema. In placebo-controlled clinical trial, dysgeusia was the most common adverse reaction reported in over 4% of subjects who received Berinert and more frequently than in placebo group. Thrombotic events have been reported in patients receiving C1 esterase inhibitor product, including Berinert, at the recommended dose as well as when used off-label or at higher-than-labeled doses.

Berinert is derived from human plasma. The risk of transmission of infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease ( CJD ) agent, cannot be completely eliminated.

Hereditary angioedema is a rare genetic disorder caused by a deficiency of C1-INH. It is inherited in an autosomal dominant manner. Symptoms of hereditary angioedema include episodes of edema, or swelling, in the face, abdomen, larynx and extremities. Patients who have abdominal attacks of hereditary angioedema can experience episodes of extreme pain, diarrhea, nausea and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if untreated, death. Diagnosis of hereditary angioedema requires a blood test to confirm low or abnormal levels of C1-INH.

Source: CSL Behring, 2012

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