You are here
Shire Provides Regulatory Progress Update for Key Pipeline Programs Targeting Rare Disease
Shire Provides Regulatory Progress Update for Key Pipeline Programs Targeting Rare Disease
- U.S. FDA Grants Orphan Drug Designation for Investigational SHP647 for Treatment of Pediatric Patients with Moderately to Severely Active Crohn's Disease
- Health Canada Grants Priority Review for Investigational Lanadelumab for Prevention of Angioedema Attacks in Patients 12 Years and Older with Hereditary Angioedema
Cambridge, Mass. - February 13, 2018 - Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Shire's investigational therapy, SHP647, for the treatment of pediatric patients with moderately to severely active Crohn's disease. Shire is preparing to conduct Phase 3 trials investigating SHP647 for the treatment of moderately to severely active Crohn's disease in adults. Pediatric study plans with SHP647 are currently under discussion with health authorities.
Health Canada has accepted Shire's request for priority review for the New Drug Submission (NDS) for lanadelumab (SHP643), an investigational therapy for prevention of angioedema attacks in patients 12 years and older with hereditary angioedema (HAE). Health Canada's review of the NDS under Priority Review is expected to be completed in the second half of 2018.
Pediatric onset Crohn's disease presents often with a more complicated disease course compared to adults and can have an impact on patients' growth, pubertal and emotional development. In the U.S., the prevalence of Crohn's disease in the pediatric population is approximately 58 in 100,000.
HAE is a rare, genetic disorder that results in recurring attacks of edema (swelling) in various parts of the body, including the abdomen, face, feet, genitals, hands and throat.,,
The swelling can be debilitating and painful, resulting in significant impact on quality of life for those individuals with HAE.
U.S. FDA Orphan Drug status is intended to advance drug development for rare diseases. The FDA provides Orphan Drug Designation to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S.
Priority review status is granted by Health Canada to a NDS for serious, life-threatening or severely debilitating diseases or conditions for which there is substantial evidence of clinical effectiveness that the drug provides an effective treatment, prevention or diagnosis of a disease or condition for which no drug is currently marketed in Canada; or a significant increase in efficacy and/or significant decrease in risk such that the overall benefit/risk profile is improved over existing therapies, preventatives or diagnostic agents for a disease or condition that is not adequately managed by a drug marketed in Canada.
SHP647 is a fully human IgG2 monoclonal antibody targeting the mucosal vascular addressin cell adhesion molecule-1 (MAdCAM-1). MAdCAM-1 plays a role in leukocyte trafficking in the GI tract and also appears to facilitate excessive lymphocyte infiltration under conditions of chronic GI inflammation. Shire is currently investigating SHP647 in Phase 3 studies for the treatment of moderately to severely active ulcerative colitis, a form of inflammatory bowel disease, in adults. Shire is also preparing to conduct Phase 3 trials investigating SHP647 for the treatment of moderately to severely active Crohn's disease in adults. Pediatric study plans with SHP647 are currently under discussion with health authorities. In November 2017, SHP647 received Orphan Drug Designation from the U.S. FDA for the treatment of pediatric patients with moderately to severely active ulcerative colitis.
Lanadelumab is an investigational fully human monoclonal antibody that specifically binds and inhibits plasma kallikrein 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.
For further information please contact:
|Christoph Brackmannfirstname.lastname@example.org||+41 795 432 359|
|Sun Kimemail@example.com||+1 617 588 8175|
|Robert Coatesfirstname.lastname@example.org||+44 203 549 0874|
|Gwen Fisheremail@example.com||+1 617 588 8607|
|Liz Kalinafirstname.lastname@example.org||+1 781 482 2713|
NOTES TO EDITORS
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.
THE "SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995
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 Company'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 Company'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;
- our ongoing strategic review of our 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, our 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 this 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 "ITEM 1A: 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.
 Ruemmele FM et al J Crohns Colitis. 2014 Oct;8(10):1179-207. doi: 10.1016/j.crohns.2014.04.005.
 Kappelman MD Dig Dis Sci. 2013 Feb;58(2):519-25. doi: 10.1007/s10620-012-2371-5.
 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.
 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.
 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.
 Kenniston JA et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J. Biol. Chem. 2014;289(34):23596-23608.
 Banerji et al. Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017; 376(8):717-728.