Jan. 16, 2013
/PRNewswire/ -- ViroPharma Incorporated (Nasdaq: VPHM) today announced the publication of data demonstrating that use of Cinryze
(C1 esterase inhibitor [human]) in pediatric patients provided relief from symptoms of hereditary angioedema (HAE) attacks and reduced the rate of attacks. HAE is a rare, severely debilitating, life-threatening genetic disorder caused by a deficiency of a human plasma protein called C1 inhibitor. The paper entitled
Nanofiltered C1-Esterase Inhibitor for The Acute Management and Prevention of Hereditary Angioedema Attacks Due to C1-Inhibitor Deficiency in Children
describes the efficacy and the safety profile of Cinryze in prevention and treatment of HAE attacks in the largest analysis of pediatric data from prospective studies of patients with HAE ever reported in medical literature. The article is in press at
The Journal of Pediatrics
and was posted online on
January 14, 2013
Cinryze is approved by the U.S. Food and Drug Administration (FDA) for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE; Cinryze is not approved to treat HAE attacks or for procedural prophylaxis. Cinryze is not approved for use in children.
According to published literature, in most patients with HAE, clinical symptoms manifest in childhood, typically between the ages of four and 11 years and may worsen during puberty. Symptoms and frequency of attacks increase during periods of intense physiologic development, such as between the ages of three and 6 years and with onset of puberty. Subcutaneous edema and recurrent abdominal pain caused by gastrointestinal edema are the most common manifestations in children. Common attack triggers include infections, emotional stress, and tissue trauma. Asphyxia is possible when angioedema involves the upper airway and can occur rapidly in children because of narrow airway diameter. Despite this, the diagnosis of HAE is often delayed until late adolescence or adulthood.
Data from the Routine Prophylaxis Placebo-Controlled Study
Four pediatric patients (ages 9 to 17 years) enrolled in and completed the pivotal prophylaxis trial. Children had a nearly 2‑fold reduction in number of HAE attacks while receiving Cinryze for prophylaxis compared with the time period during which they received placebo (mean number of attacks: 7.0 vs 13.0 over 12 weeks), consistent with the published data from the study population as a whole (6.26 vs. 12.73).