The U.S. Food and Drug Administration (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 HAE. Cinryze has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE.
Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire plc 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 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).
“This news is exciting for the HAE community because those living with HAE who are as young as 6 have a new option to help prevent attacks,” said Anthony Castaldo, President of the U.S. Hereditary Angioedema Association.
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.
Study 0624-301 was a Phase 3 multicenter, single-blind study that enrolled 12 patients aged 7 to 11 with HAE who were required to have an average of ≥1.0 angioedema attacks per month that were moderate, severe, or required acute treatment during the 12-week baseline observation period. Patients received 500 U and 1000 U of Cinryze every 3-4 days for 12 weeks. The primary efficacy endpoint was the monthly-normalized number of attacks.
Overall the safety and tolerability of Cinryze has been shown to be similar in clinical studies of pediatric, adolescent and adult patients with HAE.