February 15th, 2018
Shire Announces FDA Acceptance of sBLA for CINRYZE® (C1 esterase inhibitor [human]) for Pediatric Hereditary Angioedema Use
CAMBRIDGE, MA – February 15, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, announced today that the U.S. Food and Drug Administration (FDA) accepted the CINRYZE® (C1 esterase inhibitor [human]) 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.
CINRYZE has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE.1 HAE is a rare, genetic disorder that results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.2,3,4 It is estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.5
“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 6 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).6,7,8,9 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
CINRYZE is currently approved for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE. The active substance in CINRYZE is C1-Esterase Inhibitor (C1-INH), which raises plasma levels of C1-INH in patients with HAE, who are prone to swelling due to an underlying deficiency in C1-INH. Treatment with C1-INH addresses the underlying cause of HAE by replacing the deficient protein and helps regulate the production of bradykinin released during an attack. CINRYZE was the first C1-INH proven to help prevent swelling attacks in those living with HAE.1
Shire’s Commitment to Hereditary Angioedema
Shire is a dedicated, long-term partner to the HAE community with nearly a decade of experience supporting patients. We believe people living with HAE deserve a right-fit approach to treatment and are committed to serial innovation. Our existing portfolio of products includes a number of therapy options to help meet the needs of those living with the disease. Beyond our focus on developing novel treatments, we provide specialized services and support offerings tailored to the HAE community. Learn more at shire.com.
CINRYZE INDICATION AND IMPORTANT SAFETY INFORMATION
CINRYZE® (C1 esterase inhibitor [human]) is indicated for routine prophylaxis against angioedema attacks in adolescent and adult patients with Hereditary Angioedema (HAE).
IMPORTANT SAFETY INFORMATION
CINRYZE is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis to the product.
Hypersensitivity Reactions: Severe hypersensitivity reactions may occur during or after administration of CINRYZE. Consider treatment methods carefully, because hypersensitivity reactions and HAE attacks may have similar symptoms. In case of hypersensitivity, discontinue CINRYZE infusion and institute appropriate treatment. Have epinephrine immediately available for treatment of an acute severe hypersensitivity reaction.
Thromboembolic Events: Serious arterial and venous thromboembolic (TE) events have been reported at the recommended dose of C1 Esterase Inhibitor (Human) products, including CINRYZE, following administration in patients with HAE. Risk factors may include presence of an indwelling venous catheter/access device, prior history of thrombosis, underlying atherosclerosis, use of oral contraceptives, certain androgens, morbid obesity, and immobility. Benefits of CINRYZE for routine prophylaxis of HAE attacks should be weighed against the risks of TE events in patients with underlying risk factors. Monitor patients with known risk factors for TE events during and after CINRYZE administration.
Transmissible Infectious Agents: Because CINRYZE is made from human blood, it may carry a risk of transmitting infectious agents e.g. viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. ALL infections thought by a physician possibly to have been transmitted by CINRYZE should be reported to Shire Medical Information at 1-800-828-2088.
Adverse Reactions: The only serious adverse reaction observed in clinical studies of CINRYZE was cerebrovascular accident. The most common adverse reactions observed were headache, nausea, rash, and vomiting. Post-marketing adverse reactions include local infusion site reactions and hypersensitivity. Post-marketing thromboembolic events have been reported, including catheter-related and deep venous thrombosis, transient ischemic attack, and stroke.
For additional safety information, click for Prescribing Information and discuss with your doctor.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
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