By Brady Huggett
The beat goes on.
Similar to other steps the companies have taken in tandem, Genzyme General¿s Fabrazyme and Transkaryotic Therapies Inc.¿s Replagal both received approval ¿ orphan drug status approval ¿ from the European Commission to treat patients with Fabry disease in the 15 European Union countries.
The news wasn¿t particularly surprising, as both had received positive recommendations from the Committee for Proprietary Medicinal Products of the European Agency for the Evaluation of Medicinal Products. But whether the decision to grant both exclusivity in the EU will carry any weight with the FDA remains to be seen.
¿It was anticipated,¿ said Elise Wang, analyst with Salomon Smith Barney in New York. ¿It was in the time frame because both had received positive recommendations.¿ But what happens in Europe has little to do with how the FDA operates, Wang said.
¿I think, frankly, the FDA is quite independent,¿ she said. ¿They make their own decisions.¿
¿[The FDA] views European approvals in the corner of their eye,¿ said Thomas Dietz, analyst for Pacific Growth Equities in San Francisco. ¿But it has determined what it requires for both companies and I don¿t think there will be any influence for either company.¿
What is known is that Genzyme and TKT, both of Cambridge, Mass., will have their respective shots at EU Fabry sufferers and both are confident in their sales teams.
¿We have roughly 750 people in the European organization, both for Renagel and [our] Gaucher¿s [product], and we will do well there,¿ said David Meeker, Genzyme¿s senior vice president of therapeutics, Europe. ¿On a numbers and experience basis, that gives us an advantage.¿
Genzyme¿s established force in Europe is something Wang likes, enough for her to say she ¿tends to favor Genzyme¿ when looking at divvying up patients in the 15 countries, but said ¿both will get their share of the market because they have both done studies with physicians.¿
TKT is lining up sales troops of its own ¿ through its subsidiary called TKT Europe-5S AB ¿ to sell its first approved product.
¿[TKT Europe-5S AB] was a group of five senior members at Pharmacia [Corp., of Peapack, N.J.] that was responsible for Pharmacia¿s growth hormone program,¿ said Richard Selden, CEO and president of TKT. ¿This group was responsible for Pharmacia taking over 50 percent of that marketplace. I believe they are the most experienced niche marketers in the world. I¿m very optimistic about the infrastructure.¿
The drugs will not be cheap ¿ TKT estimates one year¿s treatment of Replagal will cost the average-sized person about $160,000. Genzyme¿s Fabrazyme will cost $100 per mg, with its product being dosed at 1 mg per kg of body weight (which could cost a similar amount per year). With estimates of Fabry patients in the EU running from 1,200 to 2,000, the revenue generation power is sizable. As the companies gear up for what Wang called a ¿marketing war¿ in the European Union, the FDA continues its work with both drugs in the States.
¿We have never set a timing expectation for when we would hear from the FDA,¿ TKT¿s Selden said. ¿We are working very closely with the FDA and we are hopeful that we will continue to make progress. It isn¿t a matter of timing, it¿s a matter of demonstrating efficacy and safety. We are an investigational drug in the U.S.¿
When asked if the FDA has all it needs to make its final decision, Genzyme¿s Meeker told BioWorld Today, ¿The best answer is that those discussions are ongoing.¿
Fabry disease is caused by a genetic disorder that leaves the body deficient in the metabolic enzyme alpha-galactosidase, and thus unable to break down some glycolipids, including globotriaosylceramide (GL-3). GL-3 accumulates in cells of key organs and the skin, causing reduced blood flow, and leading to kidney failure, stroke, cardiovascular disease, pain and numbness. Both products are designed to clear out GL-3.
The similarities of the product ¿ Fabrazyme is agalsidase beta, Replagal is agalsidase alfa ¿ stir the controversy further. Questions and remarks about the correct dosing, whether the products are separate enzymes, and issues of efficacy bounce between the companies.
¿[TKT] believes these two enzymes are different,¿ Meeker said. ¿They think they will see clinical benefit of their product. We believe that when you look at the data, I would argue, the data says they are similar proteins. If they are similar, then the question becomes what is the right dose? We get tissue clearance to a greater extent than they do.¿
¿We are very optimistic about our product,¿ Selden said. ¿When you read the European labels and when you read our label with our dosing regimen, patients experience clinical benefit without side effects such as hypersensitivity.
¿I believe that based on the data that we have submitted, we have sufficient data to demonstrate our efficacy and safety,¿ he added. ¿But on the other hand, that is the FDA¿s decision.¿
Genzyme¿s stock (NASDAQ:GENZ) rose $1.07 to close at $58.75 Friday. TKT¿s stock (NASDAQ:TKTX) jumped $2.72, or 9 percent, to end the day at $32.60.