FDA Accepts Shire's Biologics License Application (BLA) and Grants Priority Review for Lanadelumab for the Prevention of Attacks in Hereditary Angioedema (HAE) Patients

-       Lanadelumab, the first long-acting investigational monoclonal antibody in HAE, is being evaluated for the prevention of angioedema attacks in patients 12 years and older
-       HAE is a rare, genetic disorder that causes debilitating, painful and sometimes life-threatening swelling in the body[1]
-       FDA acceptance underscores Shire's leadership in rare diseases with an innovative pipeline of 15 late-stage development programs

CSMBRIDGE, MA, USA I February 23, 2018 I Shire plc (Shire) (LSE: SHP, NASDAQ: SHPG) today announced the U.S. Food and Drug Administration (FDA) accepted the Biologics License Application (BLA) and granted priority review for lanadelumab (SHP643). Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with the rare, genetic disorder, hereditary angioedema (HAE).

"Every day, patients living with HAE struggle to manage their disease - not knowing when their next attack will occur," said Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire. "Lanadelumab if approved will be the first monoclonal antibody for HAE, a serious and potentially life threating disease. Lanadelumab provides a new mechanism of action inhibiting plasma kallikrein for the prevention of HAE attacks. Lanadelumab will offer patients a new option to help control this disease with the potential to change the treatment paradigm. The FDA's decision underscores Shire's serial innovation in HAE and commitment to improving treatment options for patients."

The burden of HAE on patients is significant. On average, HAE patients take 20 days away from school or off from work per year.1, 2, 3 HAE results in recurring attacks of edema (swelling) that can be debilitating and painful in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.1, [2], [3] Attacks that obstruct the airways (asphyxiation) are potentially life-threatening.

The BLA for Shire's investigational HAE treatment is supported by data from four clinical trials, including HELP(TM), the pivotal Phase 3 efficacy and safety study, along with interim data from its extension study. HELP is the largest prevention study in HAE conducted to date, and enrolled a total of 125 patients aged 12 years and over with type I/II HAE. The HELP study demonstrated that subcutaneous administration of 300 mg lanadelumab once every two weeks resulted in an 87% reduction in the mean frequency of HAE attacks. In addition, an exploratory endpoint, which will require further confirmatory studies, showed that during the steady state stage of the trial (day 70-182) a 91% attack reduction was achieved with 8 out of 10 patients reaching an attack free state. In this study, no treatment-related serious adverse events or deaths were reported. The most common adverse event was injection site pain (29.3% placebo vs. 42.9% combined lanadelumab arms). 

"Physicians as well as patients in the HAE community are excited to see lanadelumab moving forward for FDA review because there is now the real possibility of having a new way to prevent HAE attacks," said Aleena Banerji, M.D., Massachusetts General Hospital, Boston, MA, and clinical trial investigator. "As an investigator, I am appreciative of the HAE patients who participated in the clinical trial to help advance science in a way that may transform the treatment of HAE."

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. Drugs with Priority Review designation have an accelerated review target of eight months, instead of the standard of 12 months. The FDA is expected to provide a decision on lanadelumab by August 26, 2018, based on the Prescription Drug User Fee Act V action date.

The FDA BLA acceptance of lanadelumab reinforces Shire's leadership in rare disease innovation. As the leader in rare disease drug development, Shire's commitment is global. Shire has received Priority Review and Orphan Drug Designation from the Therapeutic Goods Administration in Australia and Priority Review from Health Canada for review of this compound. In 2017 alone, Shire received Breakthrough Therapy, Orphan Drug or Fast Track designations by the FDA for five of its rare disease therapies and anticipates continued progress of key regulatory milestones for late-stage programs. Additional information can be found on shire.com.

About Lanadelumab
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein[4] and is being studied as a treatment for the prevention of angioedema attacks in patients 12 years and older with HAE. Lanadelumab is formulated for subcutaneous administration, and has a half-life of approximately 14 days in patients with HAE.[5]

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 each patient deserves 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 appropriate patients with HAE. Beyond our focus on developing novel treatments, we provide specialized services and support offerings tailored to the HAE community. Learn more at shire.com.

About Shire

Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.

We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people's lives with medicines that have a meaningful impact on patients and all who support them on their journey.

www.shire.com

[1] Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

[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] Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

[4] Kenniston JA et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J. Biol. Chem. 2014;289(34):23596-23608.

[5] Banerji et al. Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017; 376(8):717-728.

SOURCE: Shire

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