Update: Takhzyro (lanadelumab-flyo) Now FDA Approved - August 23, 2018
FDA Accepts Shire’s Biologics License Application (BLA) and Grants Priority Review for Lanadelumab for the Prevention of Attacks in Hereditary Angioedema (HAE) Patients
Cambridge, Mass. – February 23, 2018 – 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™, 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.
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein4 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.
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:
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 the 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 the 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 the 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;
Shire's ongoing strategic review of its Neuroscience franchise may distract management and employees and may not lead to improved operating performance or financial results; there can be no guarantee that, once completed, Shire's strategic review will result in any additional strategic changes beyond those that have already been announced; 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.
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 plc
Posted: February 2018
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