Fourth Quarter 2003 Replagal Sales Expected to Approximate Third Quarter Sales
CAMBRIDGE, Mass., Jan. 12 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that it will cease efforts to
seek the approval of Replagal(TM) (agalsidase alfa) enzyme replacement therapy
for Fabry disease from the U.S. Food and Drug Administration (FDA). TKT will
continue to market and sell Replagal in countries where it is approved outside
the United States, will continue its efforts to introduce Replagal in new
markets in 2004, and will continue to supply Replagal to U.S. patients in its
ongoing clinical trials.
TKT submitted a request for a Special Protocol Assessment to the FDA in
November 2003 to identify a regulatory approach that could lead to U.S.
approval of Replagal, where orphan drug exclusivity excludes it from the U.S.
market. In its response, the FDA indicated it would expect TKT to conduct a
head-to-head trial comparing Replagal to Fabrazyme in patients with Fabry
disease as the way to demonstrate either superior safety or efficacy.
"We always characterized this initiative as one that faced significant
challenges and one that was not a necessary part of our business plan. We are
nonetheless disappointed that U.S. patients will not have the benefits of
Replagal," said Michael J. Astrue, President and Chief Executive Officer of
TKT. "Given the limited availability of Fabry patients for clinical trials
and the large number of patients necessary to conduct a meaningful head-to-
head trial, we feel we should put our resources into expanding the existing
Replagal franchise and other important programs for desperately ill patients."
TKT also announced today that, based on a preliminary review of vial
shipments, it expects fourth quarter 2003 Replagal product sales to
approximate third quarter 2003 product sales, which totaled $15.2 million.
TKT also believes its cash balance at December 31, 2003 will exceed its 2003
guidance of $150 to $170 million. The company cautions that all of today's
projections are based on preliminary data.
Mr. Astrue will provide an overview of TKT's product pipeline, including
Replagal, tomorrow, January 13, 2004 at 4:30 p.m. PST (7:30 p.m. EST) at the
22nd Annual JPMorgan Health Care Conference in San Francisco. The live audio
webcast and replay of the presentation will be available in the investor
information section of TKT's website at http://www.tktx.com.
About Replagal
Replagal is a fully human alpha-galactosidase A protein, produced by
genetic engineering technology, intended for long-term treatment of Fabry
disease. It replaces the deficient alpha-galactosidase A with active enzyme
to halt or reverse the disease process. Replagal has been approved for
commercial use in 27 countries, primarily in the European Union.
About Fabry Disease
Fabry disease is a rare, inherited genetic disorder caused by deficient
activity of the lysosomal enzyme alpha-galactosidase A. It affects both males
and females, which results in premature mortality in the fourth or fifth
decade of life due to kidney disease, heart disease and stroke. An estimated
5,000 patients worldwide are affected by Fabry disease. Due to its rarity and
vast array of symptoms diagnosis is often difficult and affected men and woman
have a significantly reduced quality of life and a greatly shortened life
expectancy.
About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company committed to
developing treatments for rare diseases caused by protein deficiencies with a
core focus on lysosomal storage diseases. The company markets one product,
Replagal(TM), an enzyme replacement therapy for Fabry disease in Europe and
other countries and is developing treatments for Hunter syndrome and Gaucher
disease. TKT's research pipeline is focused on rare diseases where a
significant unmet medical need exists. TKT was founded in 1988 and is
headquartered in Cambridge, Massachusetts, with additional operations in
Europe 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, including
statements regarding Replagal, sales expectations for Replagal for the fourth
quarter of 2003 and the company's cash position as of December 31, 2003, and
other statements containing the words "believes," "anticipates," "plans,"
"expects," "will" and similar expressions. These forward-looking statements
are based on the company's opinion and expectations as of the date hereof and
are subject to risks and uncertainties that could cause actual results to
differ materially. Important factors that could cause results to differ
include, but are not limited to: whether Replagal will be approved in the
United States, if at all; whether regulatory authorities equivalent to the FDA
will approve Replagal on a timely basis, or at all; whether, if approved, this
product will achieve commercial success; whether TKT's preliminary review of
the number of vials shipped is indicative of Replagal product sales that will
be recognized in the fourth quarter of 2003; the extent to which TKT's
financial results will benefit from foreign exchange fluctuations in the
fourth quarter of 2003, if at all; and other factors set forth under the
caption "Certain Factors That May Affect Future Results" in the company's
Quarterly Report on Form 10-Q for the quarter ended September 30, 2003 which
risk factors are on file with the Securities and Exchange Commission and are
incorporated herein by reference. The company does not undertake any
obligation to update any forward-looking statements.
Replagal(TM) is a trademark of Transkaryotic Therapies, Inc.
Contact:
Justine E. Koenigsberg
Director, Corporate Communications
(617) 349-0271
SOURCE Transkaryotic Therapies, Inc.
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CONTACT:
Justine E. Koenigsberg, Director, Corporate
Communications of Transkaryotic Therapies, Inc., +1-617-349-0271
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