Dublin, Ireland – Thursday, June 21, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for CINRYZE® (C1 esterase inhibitor [human]), making it available to help prevent angioedema attacks in children aged 6 years and older with hereditary angioedema (HAE). CINRYZE has been approved in the U.S. since October 2008 for routine prophylaxis against attacks in adolescents and adults living with HAE.
HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide., The condition results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat that can be can debilitating and painful.2,, Attacks that obstruct the airways (laryngeal attacks) are potentially life-threatening due to the risk of asphyxiation.2,4,5
Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire said: “Symptoms of HAE often present in childhood with the average child experiencing their first HAE attack around the age of 10. With the FDA label expansion of CINRYZE, children as young as 6 years old living with HAE now have the first FDA approved treatment option available to help prevent attacks.”
The approval was based on data from a dedicated Phase 3 multicenter single-blind study (0624-301) that evaluated the use of CINRYZE in 12 patients living with HAE aged 7 to 11. Compared to the baseline observational period, the mean reduction in the normalized number of attacks for CINRYZE 500 U and CINRYZE 1,000 U was 71.1% and 84.5%, respectively. Both doses lessened the severity of attacks and reduced the use of acute treatment compared to baseline. The adverse reactions were headache, nausea, pyrexia (fever), and infusion site erythema (redness of the skin). None of these adverse reactions were severe, and none led to discontinuation (n=12, ages 7-11).1
“This news is exciting for the HAE community because those living with HAE who are as young as 6 have a new option to help prevent attacks,” said Anthony Castaldo, President of the U.S. Hereditary Angioedema Association.
In March 2017, CINRYZE was granted European Commission (EC) approval for the label expansion granting three new indications, including routine prevention of angioedema attacks in children (ages 6 years and above) with severe and recurrent attacks of HAE.
About the CINRYZE Pediatric Study
Study 0624-301 was a Phase 3 multicenter, single-blind study that enrolled 12 patients aged 7 to 11 with HAE who were required to have an average of greater than or equal to1.0 angioedema attacks per month that were moderate, severe, or required acute treatment during the 12-week baseline observation period. Patients received 500 U and 1000 U of CINRYZE every 3-4 days for 12 weeks. The primary efficacy endpoint was the monthly-normalized number of attacks.
Overall the safety and tolerability of CINRYZE has been shown to be similar in clinical studies of pediatric, adolescent and adult patients with HAE.
CINRYZE is currently approved for routine prophylaxis against angioedema attacks in children aged 6 years and older, 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
In Europe, CINRYZE is the first and only C1-Esterase Inhibitor (C1-INH) therapy approved for routine prevention in children, adolescents, and adults with HAE. CINRYZE is also approved for acute treatment and pre-procedure prevention of angioedema attacks.
IMPORTANT SAFETY INFORMATION (U.S. ONLY)
Contraindications: 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 may have symptoms similar to HAE attacks. 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
Adverse Reactions: The only serious adverse reaction observed in clinical studies of CINRYZE was cerebrovascular accident. The most common adverse reactions (greater than or equal to 5%) observed were headache, nausea, rash, vomiting, and fever. Post marketing adverse reactions include local infusion site reactions and hypersensitivity. Post marketing thromboembolic events have been reported, including catheter-related and deep venous thromboses, transient ischemic attack, and stroke.
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To report SUSPECTED ADVERSE REACTIONS, contact Shire Medical Information at 1-800-828-2088 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Shire’s Commitment to Hereditary Angioedema
HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.2,3 The condition results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat that can be debilitating and painful. Attacks that obstruct the airways (asphyxiation) are potentially life-threatening.2,4,5 Shire is a dedicated, long-term partner to the HAE community with a decade of experience supporting patients. We are committed to serial innovation in HAE and our portfolio of products includes a number of therapy options to help meet the individual 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.
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NOTES TO EDITORS
Shire is the global biotechnology leader serving patients with rare diseases and specialized conditions. We seek to push boundaries through discovering and delivering new possibilities for patient communities who often have few or no other champions. Relentlessly on the edge of what’s next, we are serial innovators with a diverse pipeline offering fresh thinking and new hope. Serving patients and partnering with healthcare communities in over 100 countries, we strive to be part of the entire patient journey to enable earlier diagnosis, raise standards of care, accelerate access to treatment, and support patients. Our Rare Disease and Neuroscience divisions support our diverse portfolio of therapeutic areas, including Immunology, Hematology, Genetic Diseases, Internal Medicine, Ophthalmics, Oncology, and neuropsychiatry disorders.
Championing patients is our call to action – it brings the opportunity – and responsibility – to change people’s lives.
Stephen Williams. Deputy Company Secretary, is responsible for arranging the release of this announcement.