RARE DISEASE REPORT: FDA Approves HAE Drug Berinert For Pediatric Use

July 25, 2016

The FDA has approved Berinert [C1 Esterase Inhibitor (Human)], to be used in pediatric patients to treat Hereditary Angioedema (HAE) attacks. This expansion means Berinert is the only approved HAE treatment available to patients under 12 years of age.

Clinical studies have shown that intervention with Berinert at the onset of an HAE attack brings significantly faster relief to a patient and reduces the severity of the attack.

Berinert is a plasma-derived C1 Esterase Inhibitor (Human) indicated for the treatment of acute abdominal, facial, or laryngeal attacks of hereditary angioedema (HAE) in adult and pediatric patients.

Hereditary Angioedema (HAE)

HAE is a rare genetic condition estimated to occur between 1 in 10,000 to 1 in 50,000 people.  HAE patients experience episodes of swelling in their extremities, face and abdomen, with potentially life-threatening swelling of the airway. HAE patients have a defect in the gene that controls the production of C1 inhibitor and when a person’s levels of C1-Iinhibor are low, fluid from blood vessels can leak into nearby connective tissues, causing severe pain and swelling.

There are currently 5 approved orphan drugs for treating HAE. One for preventing attacks and 4 to treat attacks.

Cinryze  [C1 esterase inhibitor (human)] is FDA-approved for preventing HAE attacks.

 [C1 esterase inhibitor (human)] iis FDA-approved to treat acute HAE attacks.

Kalbitor (ecallantide) is FDA-approved to treat acute HAE attacks.

Firazyr (icatibant) is FDA-approved to treat acute HAE attacks.

Ruconest [C1 esterase inhibitor (recombinant)] is FDA-approved to treat acute HAE attacks.

Only Berinert is approved for children under the age of 12.

(SOURCE: Rare Disease Report)

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