TKT's Replagal(TM) Enzyme Replacement Therapy for Fabry Disease Granted Marketing Approval in New Zealand and Iceland
TRANSKARYOTIC THERAPIES LOGO - TKT
Transkaryotic Therapies Inc. Logo. (PRNewsFoto)[KC]
CAMBRIDGE, MA USA 09/13/1999
CAMBRIDGE, Mass., Oct. 19 /PRNewswire/ --
Transkaryotic Therapies, Inc. (Nasdaq: TKTX) today announced that it has
received marketing approval of Replagal(TM) (agalsidase alfa) enzyme
replacement therapy for the long-term treatment of Fabry disease in both New
Zealand and Iceland. Replagal has now been approved in 18 countries
worldwide. In August 2001, Replagal was granted marketing approval in both
the fifteen countries of the European Union and Norway.
"These approvals are an important component of our goal to make Replagal
globally available to patients with Fabry disease, and we are working with
regulatory authorities worldwide to obtain additional approvals," said Richard
F Selden, M.D., Ph.D., President and Chief Executive Officer of TKT. "We
believe there will be a greater awareness of Fabry disease and, ultimately,
better diagnosis of and treatment outcomes for patients as Replagal becomes
more widely available."
The approval of Replagal in New Zealand and Iceland is based on six-month
data from two independent placebo-controlled clinical trials conducted in the
United States and the United Kingdom, as well as long-term data of up to 18
months from open-label maintenance studies. As reflected in the product
labels, these studies demonstrated multiple clinical improvements including a
reduction in neuropathic pain and reduction in the use of pain medications,
initial stabilization followed by improvement in kidney function, a reduction
in cardiac mass, and a metabolic correction of glycosphingolipid levels in
urine sediment, plasma, and kidney, heart and liver cells.
Overview of Replagal(TM) Development
TKT is committed to a broad clinical program for Replagal. In a pivotal
study conducted at the NIH, twenty-six patients participated in a 24-week,
randomized, double-blind, placebo-controlled trial. In a separate pivotal
study conducted at the Royal Free Hospital in the United Kingdom, fifteen
patients also participated in a randomized, double-blind, placebo-controlled
study. Investigators have reported that data from these pivotal trials
suggest that treatment with Replagal is effective in reducing neuropathic
pain, reducing cardiac mass and stabilizing or improving kidney function and
that treatment with Replagal is safe and well-tolerated. TKT is evaluating
Replagal in female patients and those who have received renal transplants.
Additional studies are planned, including a trial to evaluate the effect of
Replagal in pediatric patients.
About Replagal(TM) Enzyme Replacement Therapy
Replagal is a human alpha-galactosidase A produced by genetic engineering
technology in a human cell line. Patients receive 0.2 mg/kg of Replagal every
other week over a 40 minute intravenous infusion. In the United States and
Europe, many patients receive Replagal at home rather than in a hospital
setting. Approximately 200 patients are receiving Replagal worldwide, of
which approximately 150 patients are in Europe.
About Fabry Disease
Fabry disease is an inherited rare genetic disorder caused by deficient
activity of the lysosomal enzyme alpha-galactosidase A affecting both males
and females. In patients with Fabry disease, globotriaosylceramide (Gb3)
accumulates in various organs and tissues of the body due to the deficiency of
alpha-galactosidase A. Many cells are damaged by Gb3 including epithelial
cells of the kidney, myocardial cells, cells of the autonomic nervous system,
and endothelial, perithelial, and smooth muscle cells in the large vessels.
As a result, the deposits of this material can result in extreme pain, severe
kidney damage, cardiovascular disease, and stroke. Due to its rarity and vast
array of symptoms, diagnosis is often difficult and affected individuals have
a significantly reduced quality of life and a greatly shortened life
expectancy. TKT estimates that approximately 5,000 patients worldwide are
affected by Fabry disease.
About TKT
Transkaryotic Therapies, Inc. (TKT) is a biopharmaceutical company
dedicated to the development and commercialization of products based on its
three proprietary development platforms: Niche Protein(TM) products, Gene-
Activated(R) proteins, and Gene Therapy. The Company's Niche Protein product
platform is based on protein replacement for the treatment of rare genetic
diseases, a group of disorders characterized by the absence of certain
metabolic enzymes. TKT's gene activation technology is a proprietary approach
to the large-scale production of therapeutic proteins, which does not require
the cloning of genes and their subsequent insertion into non-human cell lines.
The Company's gene therapy technology, known as Transkaryotic Therapy(TM), is
focused on the commercialization of non-viral, ex vivo gene therapy products
for the long-term treatment of chronic protein deficiency states.
This press release contains forward-looking statements that involve a
number of risks and uncertainties. For this purpose, any statements contained
herein that are not statements of historical fact may be deemed to be forward-
looking statements. Without limiting the foregoing, the words, "believes,"
"anticipates," "plans," "expects," "estimates," "intends," "should," "could,"
"will," "may," and similar expressions are intended to identify forward-
looking statements. 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 set forth under the caption "Risk Factors" in the
Company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2001
which is on file with the Securities and Exchange Commission and incorporated
herein by reference. These include, without limitation, the following: (1)
whether any of the Company's Niche Protein, Gene-Activated protein or gene
therapy product candidates will advance in the clinical trial process, (2)
whether such clinical trials will proceed in a timely manner, (3) whether the
clinical trial results will warrant continued product development, (4) whether
the required regulatory filings, such as Investigational New Drug applications
and Biologics License Applications, are made in a timely manner, (5) whether
the Company's products will receive approval from the U.S. Food and Drug
Administration or equivalent foreign regulatory agencies, (6) if such products
receive approval, whether they will be successfully distributed and marketed,
(7) whether patent litigation in which the Company is involved or may become
involved are resolved in a manner adverse to the Company, (8) the effects of
competitive products on the Company's proposed products, and (9) the Company's
dependence on third parties, including collaborators, manufacturers and
distributors.
Gene-Activated(R) is a registered trademark and Niche Protein(TM),
Replagal(TM), TKT(TM), and Transkaryotic Therapy(TM) are trademarks of
Transkaryotic Therapies, Inc.
Please visit our web site at tktx.com for additional information about
Transkaryotic Therapies, Inc.
CONTACT:
Daniel E. Geffken
Senior Vice President, Finance
and Chief Financial Officer
(617) 349-0581
Click here
SOURCE Transkaryotic Therapies, Inc.
Web Site: tktx.com
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