Press Release Source: Transkaryotic Therapies, Inc.
TKT Provides Regulatory and Commercial Updates on Fabry Disease Program
Wednesday October 2, 4:05 pm ET
CAMBRIDGE, Mass., Oct. 2 /PRNewswire-FirstCall/-- Transkaryotic Therapies, Inc. (Nasdaq: TKTX - News) announced today that the postponement of the September 27, 2002 meeting of the U.S. Food and Drug Administration's ("FDA") Endocrinologic and Metabolic Drugs Advisory Committee ("Advisory Committee") will likely delay the timing of a decision on the approval of TKT's Replagal(TM) (agalsidase alfa) enzyme replacement therapy for the treatment of Fabry Disease into the first half of 2003. While it is not yet clear when the FDA will reschedule the Advisory Committee meeting, TKT is communicating regularly with the FDA in the hope that the meeting occurs as soon as possible.
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TKT continues to believe that the FDA will approve Replagal, although the FDA's review of the Replagal Biologic License Application for the postponed meeting expressed concerns regarding TKT's clinical data, particularly with respect to pain. The FDA indicated that methodological issues made the pain data uninterpretable and that data supporting the primary pain endpoint did not support approval. TKT believes that these pain data demonstrate an important benefit for patients, but has nonetheless concluded that the best approach to obtain a prompt approval for Replagal in the United States is to seek approval on the basis of its renal and cardiac data. TKT believes that an Advisory Committee meeting will allow TKT to address FDA's concerns with regard to the renal and cardiac data and demonstrate the medically compelling nature of these data. While TKT will seek approval based on these functional data, TKT's recent extensive communications with the FDA indicate that the FDA is also considering using kidney pathology data as a surrogate endpoint for a possible accelerated approval.
"We believe that Replagal is a significant product for people suffering from Fabry Disease, and we are doing everything we can to try to make sure that patients in the United States benefit from this important product as quickly as possible. We are pleased that the FDA continues to work with us in the same spirit," said Richard F Selden, M.D., Ph.D., President and Chief Executive Officer of TKT.
Commercial Update
The most recent available data from TKT Europe-5S AB, a majority-owned subsidiary of TKT located in Sweden, indicate that Replagal sales in Europe were unexpectedly slow during the summer and that sales for the third quarter will be somewhat lower than for the previous quarter sales of $8.8 million. TKT now believes that its 2002 sales will likely be below its previous guidance of $35-42 million and is expected to be in the $30-35 million range. The Company will release its quarterly results on October 30, 2002. "Sales have been lower than expected over the summer, but we are back on track for strong growth in the fourth quarter and onwards," said Mr. Bo Ahlstrand, Chief Executive Officer of TKT Europe-5S.
Conference Call and Webcast
TKT will host a conference call today, October 2, 2002 at 5:00 p.m. E.T. to discuss its Fabry Disease program. Participants may access the call by dialing 785-832-1077. The call will also be broadcast live over the Internet at www.tktx.com under the Investor Information section. A replay of this conference call will be available by dialing 973-709-2089 using access code 263244 or on the Internet at www.tktx.com under the Investor Information section.
Background Information
About Replagal(TM) Enzyme Replacement Therapy
Replagal is a human alpha-galactosidase A protein, produced by genetic
engineering technology, intended for long-term treatment of Fabry Disease.
It works by replacing the deficient alpha-galactosidase A with active enzyme
to stop or reverse the disease process. Patients receive 0.2 mg/kg of
Replagal every other week over a 40-minute intravenous infusion and in the
United States and Europe, many patients receive Replagal at home rather than
in a hospital setting. Replagal is approved for commercial use in 24
countries, including the 15 countries of the European Union. In the United
States, Replagal is an investigational product.
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, which results in premature mortality in the third or fourth decade of life due to kidney disease, heart disease and stroke. 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 severe kidney damage, cardiovascular disease, stroke, and extreme pain. 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. |
