FDA Approves Pediatric Treatment for Hereditary Angioedema

The FDA has approved C1 Esterase Inhibitor (Human) (Berinert) for treating hereditary angioedema attacks in pediatric patients, according to an announcement by CSL Behring, the company who makes the treatment. The plasma-derived C1 Esterase Inhibitor (Human) had already been approved for adult patients, and this is the first approval for pediatric patients. 

Research has indicated that treating patients with C1 Esterase Inhibitor (Human) at the beginning of a hereditary angioedema attack makes the attack less severe and leads to quicker relief. 

In clinical trials, adverse events associated with C1 Esterase Inhibitor (Human) include increased pain severity linked with hereditary angioedema attacks. In addition, more than 4% of participants in a placebo-controlled clinical trial reported dysgeusia. Thrombotic events have been reported at both the recommended dose and in off-label and higher-than-labeled doses. 

—Lauren LeBano 

Reference

U.S. FDA Approves CSL Behring's Berinert® as the First and Only Pediatric Treatment for Hereditary Angioedema (HAE) [press release]. Jersey City, NJ: PR Newswire; July 18, 2016.