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Efforts To Seek U.S. Approval for Agalsidase Halted
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 www.tktx.com.
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.
Source: Transkaryotic Therapies, Inc.