Shire Announces FDA Approval for Label Expansion of CINRYZE® for Prevention of Attacks in Pediatric HAE

Shire plc (NASDAQ:SHPG; LSE:SHP) has announced that the US Food and Drug Administration (:FDA) has approved a label expansion for CINRYZE to extend to help prevent angioedema attacks in children aged 6 and older with hereditary angioedema (HAE).

As quoted in the press release:

 CINRYZE has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE.^[1]

HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.^[2],[3] 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.^2,[4],[5] Attacks that obstruct the airways (laryngeal attacks) are potentially life-threatening due to the risk of asphyxiation.^2,4,5

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.^[6] 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 (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).¹

Click here to read the full press release.