CAMBRIDGE, Mass., July 15 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that it has received fast track
designation from the U.S. Food and Drug Administration (FDA) for iduronate-2-
sulfatase (I2S) to treat Hunter syndrome (MPS II). TKT commenced a pivotal
study with I2S in September 2003. The study, referred to as AIM (Assessment of
Iduronate-2-sulfatase in MPS II) is a twelve-month, randomized, double-blind,
placebo-controlled trial evaluating I2S in 96 patients. TKT expects top-line
results from the AIM study in the second quarter of 2005 and, if positive, the
company expects to submit applications for marketing approval in both the
United States and Europe in the second half of 2005.
"We are pleased that the FDA has given I2S this important designation,"
said Michael J. Astrue, President and Chief Executive Officer of TKT. "We are
committed to bringing this treatment to patients as quickly as possible."
About Fast Track Designation
Under the FDA Modernization Act of 1997, fast track regulations are
designed to facilitate the development of products to treat serious or life-
threatening diseases where an unmet medical need exists. Fast track
regulations are also designed to expedite the review process for designated
products, including the potential for companies to submit marketing
applications on a rolling basis.
About I2S
I2S is a human iduronate-2-sulfatase produced by genetic engineering
technology intended for long-term treatment of Hunter syndrome. The rationale
for the therapy is that I2S would replace enzyme that is deficient in patients
with Hunter syndrome and either stop or reverse disease progression. I2S has
been designated an orphan drug in both the United States and Europe and is the
only known enzyme replacement therapy in development for the treatment of
Hunter syndrome.
About Hunter Syndrome
Hunter syndrome is a genetic disorder, also referred to as MPS II. This
hereditary disorder is characterized by the body's inability to produce the
enzyme iduronate-2-sulfatase, which is essential in the continuous process of
replacing and breaking down glycosaminoglycans (GAG). As a result, GAG
remains stored in cells in the body causing progressive damage. The symptoms
of Hunter syndrome are usually not visible at birth, but usually start to
become noticeable after the first year of life. Often the first symptoms may
include hernias, frequent ear infections, runny noses, and abnormal facial
appearance. As the disease progresses, a variety of symptoms appear
including, enlarged liver and spleen, heart failure, obstructive airway
disease, sleep apnea, joint stiffness, and, in the severe form, central
nervous system involvement. In severe cases, the life expectancy for patients
with Hunter syndrome is typically 10-15 years of age. However, in the
attenuated form of the disease, patients can survive into the fifth or sixth
decade of life. TKT believes there are up to 2,000 patients worldwide
afflicted with Hunter syndrome in jurisdictions where reimbursement may be
possible.
About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company primarily
focused on researching, developing and commercializing treatments for rare
diseases caused by protein deficiencies. Within this focus, the company
markets Replagal(TM), an enzyme replacement therapy for Fabry disease, and is
developing treatments for Hunter syndrome and Gaucher disease. Outside its
focus on rare diseases, TKT intends to commercialize Dynepo(TM), its Gene-
Activated(R) erythropoietin product for anemia related to kidney disease, in
the European Union. TKT was founded in 1988 and is headquartered in Cambridge,
Massachusetts, with additional operations in Europe, Canada and Latin America.
Additional information about TKT is available on the company's website at
http://www.tktx.com.
This press release contains forward-looking statements regarding I2S, as
well as statements containing the words "believes," "anticipates," "plans,"
"expects," "estimates," "intends," "should," "could," "will," "may," and
similar expressions. There are a number of important factors that could cause
the company's actual results to differ materially from those indicated by such
forward-looking statements, including whether I2S will be safe and effective
as a treatment for Hunter syndrome; whether TKT will be able to successfully
complete clinical testing of I2S; whether I2S receives priority review;
whether the FDA will approve I2S on a timely basis, or at all; whether, if
approved, this product will achieve commercial success; whether competing
products will reduce any market opportunity that may exist for I2S and other
factors set forth under the caption "Risk Factors" in the company's Current
Report on Form 8-K filed April 29, 2004, which is on file with the Securities
and Exchange Commission and are incorporated herein by reference. While the
company may elect to update forward-looking statements at some point in the
future, the company specifically disclaims any obligation to do so, even if
its expectations change.
Gene-Activated(R) is a registered trademark and Replagal(TM) is a
trademark of Transkaryotic Therapies, Inc. Dynepo(TM) is a trademark of
Aventis Pharmaceuticals, Inc.
For More Information Contact:
Justine E. Koenigsberg
Director, Corporate Communications
(617) 349-0271
Daniella M. Lutz
Corporate Communications Specialist
(617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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CONTACT:
Justine E. Koenigsberg, Director, Corporate
Communications, +1- 617-349-0271, or Daniella M. Lutz, Corporate
Communications Specialist, +1- 617-349-0205
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