FDA accepts supplemental application to expand indication of Cinryze to children aged 6 years and older with hereditary angioedema.- Shire.

The FDA has accepted the Cinryze (C1 esterase inhibitor [human]), from Shire, supplemental Biologics License Application (sBLA) to expand the currently approved indication to include children aged 6 years and older with hereditary angioedema (HAE). The filing has received Priority Review designation from the FDA, which means Cinryze has an accelerated review target of eight months, instead of the standard of 12 months. The FDA is expected to provide a decision on the expanded indication of Cinryze by June 20, 2018, based on the Prescription Drug User Fee Act V action date.

This sBLA for Cinryze is supported by data from two open-label studies (LEVP 2006-1 and LEVP 2006-4) and two pediatric clinical studies (0624-203 and 0624-301). The pediatric studies used in this filing are the only clinical trials investigating a prophylactic therapy in the HAE pediatric population.