Dublin, Ireland – April 18, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on rare diseases, announced today that the Swiss Agency for Therapeutic Products (Swissmedic) has validated the marketing authorization application (MAA) for lanadelumab (SHP643). The validation of the MAA confirms that the lanadelumab MAA submission is complete and that the formal review process will begin. Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients 12 years and older with hereditary angioedema (HAE). Lanadelumab was also designated orphan drug status by Swissmedic.
HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide.1,2 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.1,3,4 Attacks that obstruct the airways (asphyxiation) are potentially life-threatening.1,3,4
Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire said, “Today’s announcement represents another important step forward as we continue our work to make lanadelumab available to the global HAE community. For those living with HAE, the recurring attacks of swelling can be debilitating. Lanadelumab, if approved, has the potential to change the HAE treatment landscape by directly targeting plasma kallikrein to inhibit excessive bradykinin formation, which stops the blood vessel permeability that causes these swelling attacks.”
Lanadelumab Regulatory Status
Regulatory filings are supported by data from four clinical trials, including HELP™, the pivotal Phase 3 efficacy and safety study, along with interim data from its ongoing extension study. HELP, the largest prevention study in HAE conducted to date, 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 versus placebo. In addition, an exploratory endpoint and post-hoc analysis which would require confirmatory studies, showed that during the steady state stage of the trial (day 70-182), a 91% attack reduction was achieved versus placebo, and nearly 8 out of 10 patients reached an attack free state. In this study, no treatment-related serious adverse events or deaths were reported. The most commonly reported treatment-related adverse events in patients treated with lanadelumab during the entire treatment period were injection site pain (29.3% placebo vs. 42.9 % combined lanadelumab arms), viral upper respiratory tract infection, headache, injection site erythema, injection site bruising, and dizziness. Most adverse events were mild to moderate in severity.
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein5 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.6
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 we are committed to ongoing 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.
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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.
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:
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 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.
2 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.
3 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.
4 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.
5 Kenniston JA et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J. Biol. Chem. 2014;289(34):23596-23608.
6 Banerji et al. Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017; 376(8):717-728.