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.
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.
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:
shire.
"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.
Shire PLC
shire/shire
View drug information on Elaprase.
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