FDA Approves First and Only Drug for Pediatric Hereditary Angioedema
CSL Behring’s Berinert® got approved by the U.S. Food and Drug Administration (FDA) as the first and the only pediatric treatment for the patients of Hereditary Angioedema (HAE).
Berinert is a human plasma derived C1 Esterase Inhibitor, it is generally indicated for the treatment of acute abdominal, facial, or laryngeal attacks of HAE attacks in adult and pediatric patients.
Figure 1. Berinert packaging (photo credit: berinert.com).
HAE is a rare, hereditary blood disorder which causes swelling under the skin in different parts of the body. The disease primarily caused due to deficiency of particular blood proteins, C1 esterase inhibitors due to which the fluids gets pushed into the peripheral areas of the body by small blood vessels and therefore causes swelling.
Figure 2. Mechanism of action of Berinert (photo credit: berinert.com).
Berinert is the only C1 Esterase Inhibitor concentrate approved for the treatment of the above described indications. It is being marketed by CSL Behring in Germany since more than 30 years and has also been approved and marketed in 20 European countries, United States, Argentina, Australia, Canada, Israel, Japan and South Korea.
This FDA approval made the Berinert available for HAE patients of all age groups and it became the first and the only approved therapy for HAE which is available for the patients below 12 years of age.
Clinical studies bolster the claim that Berinert can bring faster relief to a patient and reduce the severity of the attack.
“This is an important milestone for children living with HAE and their caregivers, to know that there is a FDA approved, safe and effective treatment option for children,” says Bob Repella, Executive Vice President, Commercial Operations, CSL Behring. “This expanded indication is an example of CSL Behring’s commitment to HAE and our continuing efforts to deliver on our promise to improve the care of patients living with serious medical conditions.”
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