The U.S. Food and Drug Administration (FDA) has accepted the Cinryze (C1 esterase inhibitor [human]) supplemental Biologics License Application (sBLA) to expand the currently approved indication to include children aged six years and older with 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.
Cinryze has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE.
“Adults and adolescents living with HAE have used Cinryze to help reduce the frequency and severity of attacks for nearly a decade,” said Jennifer Schranz, Global Development Lead, HAE, Shire. “Shire committed to studying the safety and efficacy of our HAE therapies in children aged six years and older because we understand the importance of this work to families in the HAE community. We look forward to working closely with the FDA in the coming months on this important review.”
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.
The FDA grants Priority Review designation to drugs that have the potential to provide significant improvements in the safety or effectiveness for the treatment, diagnosis or prevention of a serious disease.