FDA approves expanded label for Cinryze to treat children with hereditary angioedema .- Shire plc.

Shire plc announced that the FDA has approved a label expansion for Cinryze (C1 esterase inhibitor [human]), making it available to help prevent angioedema attacks in children aged 6 years and older with hereditary angioedema (HAE). Cinryze has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE.

HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide. The condition results in recurring attacks of edema (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 (laryngeal attacks) are potentially life-threatening due to the risk of asphyxiatio.

Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire said: “Symptoms of HAE often present in childhood with the average child experiencing their first HAE attack around the age of 10. 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 III multicenter single-blind study (0624-301) 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 and Cinryze 1,000 U was 71.1% and 84.5%, respectively. Both doses lessened the severity of attacks and reduced the use of acute treatment compared to baseline. The adverse reactions were headache, nausea, pyrexia (fever), and infusion site erythema (redness of the skin). None of these adverse reactions were severe, and none led to discontinuation (n=12, ages 7-11). In March 2017, Cinryze was granted European Commission (EC) 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.