BASINGSTOKE, England and CAMBRIDGE, MA, June 14 /CNW Telbec/ - Shire plc
(LSE: SHP, NASDAQ: SHPGY, TSX: SHQ) announces that Health Canada (under
priority review) has approved ELAPRASE, a human enzyme replacement therapy for
the treatment of Hunter syndrome, for sale and marketing in Canada. Hunter
syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare,
life-threatening genetic condition mainly affecting males that results from
the absence or insufficient levels of the lysosomal enzyme
iduronate-2-sulfatase. Without this enzyme, cellular waste products accumulate
in tissues and organs, which then begin to malfunction.
ELAPRASE is the first and only enzyme replacement therapy approved for
people suffering from Hunter syndrome. The product, which is given as weekly
infusions, replaces the missing or deficient enzyme that Hunter syndrome
patients fail to produce in sufficient quantities. ELAPRASE has been shown to
improve walking capacity in these patients.
ELAPRASE has been made available on a limited basis to Canadian patients
since January 2007 through Health Canada's Special Access Program (SAP) but
will now be available on a more widespread basis across the nation. Health
Canada's approval follows the July 2006 marketing approval of ELAPRASE by the
U.S. Food and Drug Administration and the January 2007 marketing authorization
of ELAPRASE by the European Commission. At the end of the first quarter 2007,
291 patients are being treated with ELAPRASE worldwide. Shire estimates that
there are approximately 2,000 patients worldwide afflicted with Hunter
syndrome in areas where reimbursement may be possible.
"Health Canada's approval of ELAPRASE is another important step in
bringing this much-needed treatment to Hunter syndrome patients around the
world," said Matthew Emmens, chief executive officer of Shire. "Also at this
time we want to thank the Canadian patients for their participation in the
ELAPRASE clinical trials; without their commitment and determination, we would
not have been able to bring this treatment to Canada and others would continue
to suffer the debilitating symptoms of Hunter syndrome."
According to Dr. Lorne Clarke, Medical Director of the Provincial Medical
Genetics Program and researcher at the University of British Columbia, "The
approval of ELAPRASE is an exciting advancement. There is potential to make a
significant improvement in this progressive disorder by treating patients
Clinical Trial Results
A 53-week, randomized, double-blind, placebo-controlled Phase II/III
trial demonstrated that ELAPRASE provides clinically important benefits to
Hunter syndrome patients. The primary efficacy endpoint of the trial was a
composite analysis of changes from baseline in two clinical measures: a
6-minute walk test and percent predicted forced vital capacity. Shire is
pleased to report that this endpoint achieved statistical significance
compared to placebo. Also, after one year of treatment, patients receiving
weekly infusions of ELAPRASE experienced a significant mean increase in the
distance walked in six minutes of 35 meters compared to patients receiving
placebo. The change in percent predicted forced vital capacity was not
statistically significant compared to placebo.
Anaphylactoid reactions, which have the potential to be life threatening,
have been observed in some patients treated with ELAPRASE. Patients with
compromised respiratory function or acute respiratory disease may be at risk
of serious exacerbation of their respiratory dysfunction due to infusion
related reactions. These patients require additional monitoring. Late-emergent
anaphylactoid reactions have been observed after ELAPRASE administration.
Patients who have experienced severe and refractory anaphylactoid reactions
may require prolonged observation times. Due to the potential for severe
infusion reactions appropriate medical support measures should be readily
available when ELAPRASE is administered.
In all phases of clinical study for ELAPRASE, 11 patients experienced
anaphylactoid reactions during 19 of 8,274 infusions (0.2%) and no patients
discontinued treatment permanently as a result of an infusion reaction. The
most common adverse events observed in (greater than)30% of patients during
the Phase II/III trial were pyrexia, headache and arthralgia.
Fifty percent of patients across all studies (53 of 106) developed
anti-idursulfase IgG antibodies.
Adverse reactions that were reported during the 53-week
placebo-controlled study were almost all mild to moderate in severity.
Studies have not been performed in patients under 5 or over age 65.
ELAPRASE is a purified form of the lysosomal enzyme iduronate-2-sulfatase
and is produced by recombinant DNA technology in a human cell line.
Shire Human Genetic Therapies is actively tracking health data among
individuals affected by Hunter syndrome as part of the company's long-term
outcome survey, called the Hunter Outcome Survey (HOS). HOS is designed to
support the gathering, analysis, reporting and sharing of data from around the
world about Hunter syndrome. Shire believes that the inclusion of all people
affected by Hunter syndrome, whether on therapy or not, and the analysis and
dissemination of the information gathered, will allow for better understanding
of Hunter syndrome and disease education on a global scale.
More information about ELAPRASE and Hunter syndrome is available through
Shire Human Genetic Therapies in Canada at 1-416-322-2886.
About Hunter Syndrome
Hunter syndrome (MPS II) is a serious genetic disorder mainly affecting
males that interferes with the body's ability to break down and recycle waste
substances called mucopolysaccharides, also known as glycosaminoglycans or
GAG. Hunter syndrome is one of several related lysosomal storage diseases.
In Hunter syndrome, cumulative build-up of GAG in cells throughout the
body interferes with the way certain tissues and organs function, leading to
severe clinical complications and early mortality. Physical manifestations for
some people with Hunter syndrome may include distinct facial features, a large
head and an enlarged abdomen. People with Hunter syndrome may also experience
hearing loss, thickening of the heart valves leading to a decline in cardiac
function, obstructive airway disease, sleep apnea, and enlargement of the
liver and spleen. In some cases, central nervous system involvement leads to
progressive neurologic decline.
Notes to Editors
Shire's strategic goal is to become the leading specialty
biopharmaceutical company that focuses on meeting the needs of the specialist
physician. Shire focuses its business on attention deficit and hyperactivity
disorder (ADHD), human genetic therapies (HGT), gastrointestinal (GI) and
renal diseases. The structure is sufficiently flexible to allow Shire to
target new therapeutic areas to the extent opportunities arise through
acquisitions. Shire believes that a carefully selected portfolio of products
with a strategically aligned and relatively small-scale sales force will
deliver strong results.
Shire's focused strategy is to develop and market products for specialty
physicians. Shire's in-licensing, merger and acquisition efforts are focused
on products in niche markets with strong intellectual property protection
either in the US or Europe.
For further information on Shire, please visit the Company's website:
"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM
ACT OF 1995
Statements included herein that are not historical facts 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
affected. The risks and uncertainties include, but are not limited to, risks
associated with: the inherent uncertainty of pharmaceutical research, product
development, manufacturing and commercialization; the impact of competitive
products, including, but not limited to the impact of those on Shire's
Attention Deficit and Hyperactivity Disorder (ADHD) franchise; patents,
including but not limited to, legal challenges relating to Shire's ADHD
franchise; government regulation and approval, including but not limited to
the expected product approval dates of SPD503 (guanfacine extended release)
(ADHD) and SPD465 (extended release triple-bead mixed amphetamine salts)
(ADHD); Shire's ability to secure new products for commercialization and/or
development; Shire's ability to benefit from its acquisition of New River
Pharmaceuticals Inc.; and other risks and uncertainties detailed from time to
time in Shire plc's filings with the Securities and Exchange Commission,
particularly Shire plc's Annual Report on Form 10-K for the year ended
December 31, 2006.
For further information:
For further information: Investor Relations: Clea Rosenfeld (Rest of the
World), +44-1256-894-160; Eric Rojas (North America), (484) 595-8252; Media:
Jessica Mann (Rest of the World), +44-1256-894-280; Matthew Cabrey (North
America), (484) 595-8248; Charles Pitts - (Oromedia) (CANADA) (514) 697-9111,