The U.S. Food and Drug Administration (FDA) has recently approved a new treatment for a rare genetic disorder called hereditary angioedema (HAE). The treatment, called Berinert, is manufactured by CSL Behring and is the first and only therapy approved for the treatment of acute attacks of HAE in pediatric patients.

HAE is a rare genetic disorder that affects about 1 in 10,000 to 1 in 50,000 people worldwide. It is characterized by recurrent episodes of swelling, or edema, in various parts of the body including the hands, feet, face, and airways. These attacks can be severe and potentially life-threatening, especially when the swelling occurs in the airways, which can lead to difficulty breathing and asphyxiation.

Berinert is a human plasma-derived C1 esterase inhibitor (C1-INH) that works by replacing the deficient or malfunctioning C1-INH protein in patients with HAE. The treatment is administered through intravenous infusion and can provide rapid relief of symptoms within minutes.

The FDA's approval of Berinert for pediatric patients with HAE was based on the results of a clinical trial that involved 27 patients between the ages of 6 and 17 years. The trial showed that Berinert was safe and effective in treating acute attacks of HAE in this patient population.

"We are pleased to provide this important treatment option for pediatric patients with hereditary angioedema," said Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research. "This approval represents an important step forward in our efforts to provide safe and effective therapies for rare genetic disorders."

The approval of Berinert for pediatric patients with HAE is welcome news for the HAE community, as there are currently no FDA-approved treatments for this patient population. Prior to the approval of Berinert, pediatric patients with HAE were treated with off-label use of existing therapies approved for adults, which could result in suboptimal dosing or adverse effects.

"We are thrilled with the FDA's decision to approve Berinert for use in pediatric patients with HAE," said Anthony J. Castaldo, president and CEO of the US Hereditary Angioedema Association. "This approval provides a much-needed treatment option for pediatric patients with HAE and represents a significant step forward in improving the lives of those affected by this rare disease."

In conclusion, the approval of Berinert for pediatric patients with HAE is a significant development in the treatment of this rare genetic disorder. This new treatment option provides much-needed relief for pediatric patients with HAE and represents an important step forward in improving the lives of those affected by this disease.

https://www.fda.gov/news-events/press-announcements/fda-approves-treatment-rare-genetic-disorder.