FDA Expands Approved Use of Shire’s Cinryze to Include Prevention of Pediatric HAE Attacks

Rare Daily Staff

The U.S. Food and Drug Administration has approved a label expansion for Shire’s C1 esterase inhibitor Cinryze to include prevention of attacks in children aged 6 and older with hereditary angioedema.

HAE is a rare, genetic disorder that results in recurring attacks of swelling in various parts of the body, including the abdomen, face, feet, genitals, hands and throat that can be can debilitating and painful. Attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.

Cinryze has been approved in the United States since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE. In March 2017, Cinryze was granted European Commission approval for the label expansion granting three new indications, including routine prevention of angioedema attacks in children ages 6 years and above with severe and recurrent attacks of HAE.

“Symptoms of HAE often present in childhood with the average child experiencing their first HAE attack around the age of 10,” said Andreas Busch, executive vice president and head of research and development at Shire. “With the FDA label expansion of Cinryze, children as young as 6 years old living with HAE now have the first FDA approved treatment option available to help prevent attacks.”

The approval was based on data from a dedicated phase 3 multicenter single-blind study that evaluated the use of Cinryze in 12 patients living with HAE aged 7 to 11. Compared to the baseline observational period, the mean reduction in the normalized number of attacks for Cinryze 500 U was 71.1 percent and Cinryze 1,000 was 84.5 percent.

Both doses lessened the severity of attacks and reduced the use of acute treatment compared to baseline. The adverse reactions were headache, nausea, fever, and infusion site redness of the skin. None of these adverse reactions were severe, and none led to discontinuation.

June 21, 2018
Photo: Andreas Busch, executive vice president and head of research and development at Shire