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CHMP Recommends EU Marketing Authorisation of lanadelumab for the Prevention of HAE Attacks


  • If approved, lanadelumab would be the first monoclonal antibody for the preventive treatment of Hereditary Angioedema (HAE) available in the European Union
  • In the pivotal study, patients taking lanadelumab 300 mg every 2 weeks had an 87% reduction in mean monthly attacks vs. placebo (adjusted P<0.001)
  • Lanadelumab was recently approved in the U.S. and Canada for the prevention of attacks of HAE in adolescents and adults (12 years of age and older)

Dublin, Ireland – 19 October 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of marketing authorisation of lanadelumab injection for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. If approved, lanadelumab will be a first-of-its-kind, fully human monoclonal antibody (mAb) available in the EU that inhibits the activity of plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to help prevent attacks.1

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 oedema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat, that can be debilitating and painful. Laryngeal attacks that obstruct the airways are potentially life-threatening due to the risk of asphyxiation.2,4,5

“This positive opinion marks an important step towards providing adults and adolescents living with HAE in Europe a first-of-its-kind monoclonal antibody treatment option to help prevent attacks,” said Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire. “We are excited about the future potential of lanadelumab in helping to address the needs of those living with this chronic and unpredictable disease.”

The positive opinion is supported by data from the Phase III HELP (Hereditary Angioedema Long-term Prophylaxis) Study, the largest randomised controlled prevention study conducted to date in HAE, which evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 weeks in 125 patients 12 years of age or older with HAE.6

Lanadelumab was previously granted accelerated assessment by the EMA, reducing the number of evaluation days required from 210 to 150. The CHMP's positive opinion will be reviewed by the European Commission, which has the authority to grant marketing authorisation in the EU.

Lanadelumab received approval for the prevention of HAE attacks in patient 12 years and older in the U.S. on 23 August 2018 and Canada on 19 September 2018, under the brand name TAKHZYRO.1,7

Supportive Data
The CHMP submission was based on outcomes of the HELP Study, a multicentre, randomised, double-blind, placebo-controlled parallel group trial that evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 weeks in 125 patients 12 years of age or older with HAE.6

The primary endpoint of the HELP Study was the number of investigator-confirmed HAE attacks over the entire 26-week study duration. Lanadelumab demonstrated that subcutaneous injections every two or four weeks reduced the mean monthly number of attacks across all three lanadelumab treatment arms studied: 300 mg every two weeks, 300 mg every four weeks, and 150 mg of lanadelumab every four weeks. At 300 mg every two weeks, lanadelumab reduced the number of mean monthly HAE attacks by 87% vs. placebo (adjusted P<0.001).6

Overall, each lanadelumab treatment arm demonstrated statistically significant attack rate reductions compared with placebo for all secondary efficacy endpoints (adjusted P<0.001 for all comparisons). Patients taking lanadelumab 300 mg every 2 weeks had 83% fewer moderate or severe attacks and 87% fewer attacks that needed on-demand treatment. A pre-specified, exploratory analysis showed that 44% of patients (n=27) receiving lanadelumab 300 mg every two weeks had zero attacks compared to placebo (2%, n=41) for the 26-week treatment period.1 Additionally, in a post hoc sensitivity analysis of the steady state period from Day 70 to Day 182, 77% of patients (n=26) treated with lanadelumab in the same dosage arm of the trial were attack-free compared to placebo (3%, n=37).8

Relevant Safety Information from the HELP Study
No serious Treatment Emerging Adverse Events (TEAEs) or deaths were reported.

The most common TEAE was HAE attack. The most commonly reported adverse events, excluding events related to HAE attacks, were injection site pain (42.9%), followed by upper respiratory infection (23.8%), headache (20.2%) and injection site erythema (9.5%). The majority of TEAEs were mild to moderate in severity. TEAEs resulted in discontinuation for 1 patient from the 300 mg every four weeks lanadelumab arm (ALT/AST elevation) and 2 from the placebo arm (tension headache, HAE attack).

About lanadelumab
Lanadelumab, a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity, is currently being evaluated by the EMA for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. Lanadelumab is formulated for subcutaneous administration and has a half-life of approximately two weeks in patients with HAE.6 Lanadelumab is intended for self-administration or administration by a caregiver, only after training by a healthcare professional.

For full U.S. and Canada Prescribing Information, including the approved indications and important safety information, please visit US Prescribing Information and the Canadian Product Monograph.

Shire’s Commitment to Hereditary Angioedema
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 specialised services and support offerings tailored to the HAE community. Learn more at shire.com.

For further information please contact:

Investor Relations

Christoph Brackmannchristoph.brackmann@shire.com+41 41 288 4129
Sun Kimsun.kim@shire.com+1 617 588 8175
Scott Burrowsscott.burrows@shire.com+41 41 288 4195
Media
Katie Joycekjoyce@shire.com+1 781 482 2779

NOTES TO EDITORS

About Shire

Shire is the global biotechnology leader serving patients with rare diseases and specialised 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 diverse portfolio of therapeutic areas includes Immunology, Haematology, Genetic Diseases, Neuroscience, Internal Medicine, and Ophthalmics.

Championing patients is our call to action - it brings the opportunity - and responsibility - to change people’s lives.

www.shire.com

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Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, projected revenues, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

  • Shire’s products may not be a commercial success;
  • increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire’s future revenues, financial condition and results of operations;
  • Shire depends on third parties to supply certain inputs and services critical to its operations including certain inputs, services and ingredients critical to its manufacturing processes. Any disruption to the supply chain for any of Shire’s products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
  • the manufacture of Shire’s products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
  • the nature of producing plasma-based therapies may prevent Shire from timely responding to market forces and effectively managing its production capacity;
  • Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
  • the actions of certain customers could affect Shire’s ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire’s revenues, financial conditions or results of operations;
  • failure to comply with laws and regulations governing the sales and marketing of its products could materially impact Shire’s revenues and profitability;
  • Shire’s products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
  • Shire’s patented products are subject to significant competition from generics;
  • adverse outcomes in legal matters, tax audits and other disputes, including Shire’s ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
  • Shire may fail to obtain, maintain, enforce or defend the intellectual property rights required to conduct its business;
  • Shire faces intense competition for highly qualified personnel from other companies and organizations;
  • failure to successfully execute or attain strategic objectives from Shire’s acquisitions and growth strategy may adversely affect Shire’s financial condition and results of operations;
  • Shire’s growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
  • a slowdown of global economic growth, or economic instability of countries in which Shire does business, could have negative consequences for Shire’s business and increase the risk of non-payment by Shire’s customers;
  • changes in foreign currency exchange rates and interest rates could have a material adverse effect on Shire’s operating results and liquidity;
  • Shire is subject to evolving and complex tax laws, which may result in additional liabilities that may adversely affect Shire’s financial condition or results of operations;
  • if a marketed product fails to work effectively or causes adverse side effects, this could result in damage to Shire’s reputation, the withdrawal of the product and legal action against Shire;
  • Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire’s revenues, financial condition or results of operations;
  • Shire faces risks relating to the expected exit of the United Kingdom from the European Union;
  • Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which has increased its borrowing costs and may decrease its business flexibility;
  • the potential uncertainty among our employees, customers, suppliers, and other business partners resulting from the announcement by Takeda Pharmaceutical Company Limited on May 8, 2018 of a recommended offer for Shire under the UK Takeover Code; and

a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM1A: Risk Factors”, and in Shire’s subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

1 TAKHZYRO (lanadelumab-flyo) injection prescribing information. Lexington, MA: Shire LLC; 2018.
2 Cicardi M, Bork K, Caballero T, et al, on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012; 67(2):147-157.
3 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.
4 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.
5 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.
6 Banerji A. Lanadelumab for prevention of attacks in hereditary angioedema: results from the phase 3 HELP study. American College of Allergy, Asthma and Immunology Meeting. 2017, 74: 1-18.
7 TAKHZYRO (lanadelumab injection) Product Monograph. Toronto, ON: Shire Pharma Canada ULC; 2018.
8 Maurer M, et al. was presented at the 2018 European Academy of Allergy and Clinical Immunology (EAACI) Congress, 26–30 May 2018, Munich, Germany.

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