The Food and Drug Administration (FDA) has approved an expanded indication for Cinryze (C1 esterase inhibitor [human]; Shire) to include pediatric patients 6 years of age and older with hereditary angioedema (HAE) for routine prophylaxis against angioedema attacks. The treatment was originally approved in 2008 for adult and adolescent patients with HAE.
The approval was based on data from a randomized, single-blind, multicenter, dose-ranging, crossover study which evaluated the safety and efficacy of Cinryze in 12 pediatric patients (7 to 11 years old). Patients received Cinryze 500 U and Cinryze 1000 U every 3 to 4 days for 12 weeks.
Compared to the baseline observational period, a reduction in the normalized number of angioedema attacks was observed for both Cinryze 500U and Cinryze 1000U (mean absolute reduction in number of HAE attacks: 2.6, 3.0 respectively; mean % reduction in HAE attacks: 71.1% and 84.5%, respectively). In addition, both doses lessened the severity of attacks and reduced the use of acute treatment compared with baseline.
“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 US Hereditary Angioedema Association.
Cinryze is supplied as a lyophilized powder in single-use vials that contain 500 Units for reconstitution.
For more information visit Cinryze.com.