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News from Shire.com June 14, 2007
Shire’s ELAPRASE™ (idursulfase)
Approved by Health Canada for Treatment of Hunter Syndrome
14 Jun 2007 - Basingstoke, UK and Cambridge MA, US – June 14, 2007 –
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 early.”
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.
Safety Data
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 >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.
About ELAPRASE
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.
further information please contact:
Investor Relations:
Cléa Rosenfeld (Rest of the World) -+44 1256 894 160
Eric Rojas (North America) - +1 484 595 8252
Media :
Jessica Mann (Rest of the World) - +44 1256 894 280
Matthew Cabrey (North America) - +1 484 595 8248
Notes to editors
SHIRE PLC
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:
www.shire.com
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