By Brady Huggett
Genzyme General received a second complete response letter from the FDA to its biologics license application to market Fabrazyme in the United States, and said the letter didn¿t contain any surprises.
¿The letter that we received from the FDA raised no new issues and we are confident that we will be able to gather the necessary information that the FDA needs to fully evaluate our BLA for Fabrazyme and submit it in a timely way,¿ said Bo Piela, spokesman for Genzyme. ¿We have already begun preparing our response.¿
Genzyme, of Cambridge, Mass., submitted its BLA to treat Fabry¿s disease patients in June 2000 and received an initial complete response letter in December 2000. Genzyme submitted its response in April and said it would respond in a ¿timely manner¿ to this letter as well. (See BioWorld Today, June 26, 2000, and Dec. 28, 2000.)
Due to competitive reasons, the company would not comment further on the letter¿s contents.
The competition in this case is Transkaryotic Therapies Inc., also of Cambridge, and its product for Fabry¿s disease, Replagal. TKT also has a biologics license application under review by the FDA and both companies are seeking orphan drug status for their products in the United States. The products have both been cleared for marketing in the European Union. (See BioWorld Today, July 6, 2001, and Aug. 6, 2001.)
Analysts¿ feelings on the letter were mixed. Michael King, of Robertson Stephens in San Francisco, commented on the letter in a research note, saying: ¿We understand no new issues were raised and that the company likely has the necessary information on hand. Therefore, we continue to expect FDA approval in [the second half of 2002].¿
Bill Tanner, analyst for SG Cowen Securities Corp. in New York, took an opposite view.
¿We think [the letter] is more onerous than pushing it back a bit,¿ he said. ¿I don¿t think we get the feeling the drug is imminently approvable.¿ Tanner, who does list Genzyme as a strong buy, feels there is a chance the FDA is seeking information concerning what he called ¿a clinically relevant endpoint.¿
He said the company¿s continued growth will be fueled by its product for Type I Gaucher¿s disease, Cerezyme, and Renagel, its phosphate binder for patients with end-stage renal disease on hemodialysis, rather than Fabrazyme. Renagel recorded impressive growth in sales for the quarter ended Sept. 30 and helped the company beat consensus estimates for earnings per share. (See BioWorld Today, Oct. 19, 2001.)
Genzyme¿s stock (NASDAQ:GENZ) rose $1.92 Monday to close at $48.70.