FDA Approves Combo Kit for Easier At-home Use of Berinert
The U.S. Food and Drug Administration (FDA) has approved a new convenience administration kit for people using Berinert, an at-home infusion treatment of acute hereditary angioedema (HAE) attacks in children and adults.
This kit is designed to provide all the components needed for at-home treatment, making Berinert’s use easier for patients and lessening the burden on specialty pharmacies that supply kit materials separately.
Included in the new combination package will be a 10 mL silicone-free syringe, an intravenous (IV) administration set, and a butterfly needle. The kit is expected to become commercially available in the U.S. by the close of September.
“HAE affects my life daily; with the concerns and stress of not only maintaining my own health as a patient, but also that of my child who also has HAE,” Machelle, an HAE patient, said in a press release. “Regardless of being on a preventive therapy or not, being prepared to rapidly treat an HAE attack is of utmost importance.”
In people with HAE, a protein called C1 esterase inhibitor (C1-INH), which normally blocks the activity of other proteins involved in inflammation and blood clotting, is either missing or not working as it should.
In the absence of functional C1-INH, the levels of a pro-inflammatory molecule called bradykinin rise to unhealthy levels, causing blood vessels to become leaky and fluid to accumulate in surrounding tissues, resulting in swelling.
Marketed by CSL Behring, Berinert is made up of a concentrate of C1-INH derived from healthy donors. It is currently the only C1-INH replacement therapy approved to treat acute HAE attacks of the abdomen, face, and throat in both adults and children.
Studies have shown that self-injecting Berinert is safe, following training by a healthcare provider. Without an at-home option, receiving an intravenous, or into-the-vein, medication often requires hospital admission, potentially delaying treatment and allowing symptoms — and patient outcomes — to worsen.
The World Allergy Organization’s Guidelines for the Management of HAE recommend that patients maintain enough medication to treat two attacks, and that they carry on-demand medication, such as Berinert, at all times.
In addition to treating acute attacks, Berinert has also been used as a short- and long-term therapy to prevent HAE attacks in some patients, although its safety and effectiveness in this particular setting is not yet established.
Berinert was first licensed in Germany in 1979. It is now marketed in over 20 European countries, as well as in the U.S., Argentina, Australia, Canada, Israel, Japan and South Korea.