TKT Granted Orphan Drug Status for Hunter Syndrome Treatment in Both Europe And The United States
CAMBRIDGE, Mass., Jan 3, 2002 /PRNewswire via COMTEX/ -- Transkaryotic Therapies, Inc. (Nasdaq: TKTX chart, msgs) today announced that the Company's Niche Protein(R) product, iduronate-2-sulfatase (I2S), an enzyme replacement therapy for the treatment of Hunter syndrome, has been designated an orphan drug in both Europe and the United States.
Generally, if a product with orphan drug designation subsequently receives the first marketing approval for the designated indication, the product is entitled to orphan drug exclusivity. In the European Union, orphan drug designation grants up to ten years of marketing exclusivity for serious disorders affecting not more than 185,000 people. In the United States, orphan drug designation provides up to seven years of marketing exclusivity for disorders affecting less than 200,000 people. Therefore, if TKT's I2S product is the first product approved by the European Commission and the U.S. Food and Drug Administration (FDA) for the treatment of Hunter syndrome, TKT will be entitled to marketing exclusivity in the European Union and the United States, respectively.
"There is a tremendous medical need to develop a treatment for this devastating disorder, and our ultimate goal is to make I2S enzyme replacement therapy available to patients worldwide," said Michael J. Astrue, Senior Vice President, Administration and General Counsel of TKT. "Orphan drug designation is an important component of our development strategy for many of our Niche Protein products to treat rare diseases, and we are pleased with the European Commission and FDA's decision to designate our I2S product an orphan drug in Europe and the United States."
In 2001, TKT initiated a twelve patient Phase I/II clinical trial to assess the safety, pharmacodynamic and pharmacokinetic profiles, and clinical activity of I2S enzyme replacement therapy in patients with Hunter syndrome. In the dose-escalating study, patients have been randomized to receive I2S or placebo for six months. The study, being conducted at the University of North Carolina at Chapel Hill, is fully enrolled and the first cohort of patients completed treatment and initiated maintenance dosing. TKT expects the Phase I/II trial will be completed during the first half of 2002.
About Hunter Syndrome
Hunter syndrome is an inherited rare genetic disorder caused by deficient activity of I2S. I2S is present in the lysosomes of cells, where it helps break down large biomolecules called glycosaminoglycans. Patients affected by Hunter syndrome suffer from a variety of symptoms including skeletal deformities, obstructive airway disease, cardiac failure, and, in severe cases, neurologic decline can occur. TKT estimates that approximately 5,000 patients worldwide are affected by Hunter syndrome. In severe cases, the life expectancy for patients with Hunter syndrome is typically 10-15 years of age. However, in mild cases, patients can survive into the fifth or sixth decade of life. Currently, there is no effective treatment.
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(R) 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. 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 which are set forth under the caption "Risk Factors" in the Company's Current Report on Form 8-K filed December 21, 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. In addition, any forward-looking statements represent the Company's expectations only as of the date hereof and should not be relied upon as representing the Company's expectations as of any subsequent date. 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), Niche Protein(R), and TKT(R) are registered trademarks and Transkaryotic Therapy(TM) is a trademark of Transkaryotic Therapies, Inc.
Please visit our web site at www.tktx.com for additional information about Transkaryotic Therapies, Inc.
CONTACT:
Justine E. Koenigsberg
Director, Corporate Communications
(617) 349-0271
Source: Transkaryotic Therapies, Inc.
Contact:
Justine E. Koenigsberg, Director, Corporate Communications of
TKT, +1-617-349-0271
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