VPHM Initiates Mid-Stage Study - Analyst Blog
Recently, ViroPharma Inc. (VPHM) announced the initiation of a mid-stage study to evaluate Cinryze (C1 Esterase Inhibitor (Human)) for treating acute angioedema attacks in children (n=12) suffering from hereditary angioedema (HAE).
A child has an even probability of being affected with hereditary angioedema if a parent suffers from HAE since it is an autosomal dominant disease. HAE is a rare, severely debilitating, fatal genetic disease caused by the deficiency of C1 inhibitor, a human plasma protein.
The design of the single-dose, open-label study was agreed with the Pediatric Committee of the European Medicines Agency. The agreement was part of the Pediatric Investigational Plan approved before ViroPharma’s submission of a Marketing Authorization Application (MAA) for European approval of Cinryze. The company is seeking European approval of the drug for the prevention as well as treatment of acute attacks of hereditary angioedema.
The study will evaluate the safety and tolerability of Cinryze apart from its efficacy. The primary assessment regarding the drug’s effectiveness will be based on clinical response observed within 4 hours of dosing.
Cinryze received orphan medicinal product status in the European Union in 2009. The designation in the EU is given to candidates intended to treat diseases that affect less than five in 10,000 patients.
Cinryze received approval from the U.S. Food and Drug Administration (FDA) in October 2008 for routine prophylaxis against attacks of angioedema in adolescents and adults suffering from HAE. However, the drug is not approved in the US for acute treatment of attacks or in HAE patients aged below 12 years.
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