By Matthew Willett
Transkaryotic Therapies Inc., in a race with Genzyme General for the approval and commercialization of a Fabry disease treatment, disclosed positive results from a trial of Replagal, its enzyme replacement therapy, at a conference in Vienna, Austria.
The data presented at the 10th International Congress of Human Genetics indicated Replagal therapy reduced cardiac mass and corrected the metabolic defect of Fabry disease. The therapy also appeared safe and well tolerated.
The data disclosed Friday from a randomized, double-blind, placebo-controlled, pivotal Phase II trial assessed Replagal¿s efficacy over six months in 15 patients. Patients in the treatment group, TKT reported, had a statistically significant reduction in heart mass of 4.2 percent compared to the control group¿s increase of 8.8 percent.
Patients in the treatment group also exhibited a reduction in globtriaosylceramide storage in the heart, plasma and urine.
TKT remains deadlocked with Genzyme Corp., of Cambridge, Mass., in the race to approval for the indication. In January both companies were gathering more data for the FDA.
Genzyme filed its BLA for Fabrazyme (agalsidase beta) June 23. TKT, also of Cambridge, filed its BLA for Replagal (alpha-galactosidase) June 16. The two companies are involved in patent litigation over the products, both of which have been accepted for priority review and potential orphan drug status. (See BioWorld Today, June 19, 2000, and June 26, 2000.)
A TKT representative told BioWorld Today the company couldn¿t comment on the additional data requested on Replagal by the FDA or the litigation pending between the companies.
Meanwhile, Genzyme expects its Fabry disease therapy, Fabrazyme, to receive marketing approval in the U.S. and in Europe this year ¿ within three months in Europe and in the U.S. slightly later than that. (See BioWorld Today, May 3, 2001.)
Genzyme¿s corporate communications director, Bo Piela, said enrollment in the Phase IV study of Fabrazyme began in January.
¿We¿ve submitted our response to the FDA, and we¿re continuing to make good progress in enrolling patients in the Phase IV trial,¿ Piela told BioWorld Today. ¿We did get the CPMP [Committee for Proprietary Medicinal Products of the European Agency for the Evaluation of Medicinal Products] recommendation for approval in March, recommending marketing approval for Fabrazyme, and we¿re awaiting a decision by the European Commission, which is expected to come by mid-summer.¿
TKT has also received a positive recommendation from the EU¿s CPMP for Replagal, it reported late last month.
Piela said the litigation between the companies is slated for trial in March 2002.
TKT¿s stock (NASDAQ:TKTX) rose 20 cents Friday, closing at $28.90. Genzyme¿s stock (NASDAQ:GENZ) fell $1.53 on Friday, closing at $106.34.