Phase III clinical trial results on Genzyme Corp.'s recombinantglucocerebrosidase (r-GCR) for treating Gaucher's disease werereported Tuesday at the American Pediatric Society/TheSociety for Pediatric Research Meeting in Washington, D.C.
The pivotal trial was a double-blind, randomized studycomparing the effects of r-GCR with Ceredase (Genzyme's FDA-approved natural version of the enzyme, which is derived fromhuman placenta) in 30 patients with Gaucher's disease over aperiod of six months.
The Cambridge, Mass., company announced late Tuesday thatdata from this study demonstrate that r-GCR is both safe andeffective, and that parallel and equivalent improvementsoccurred in all disease parameters in comparison to Ceredase.
"When comparing r-GCR with Ceredase there were nosignificant differences in the decrease of hepatic or splenicvolume observed or in the improvement in anemia orthrombocytopenia," said Norman Barton of the NationalInstitutes of Health, one of the trial's clinical investigators.
The FDA approved the Phase III trials protocol in lateNovember 1991. Genzyme (NASDAQ:GENZ) intends to file a newdrug application (NDA) based on the data from this trial soon,according to spokeswoman Donna LaVoie.
Although the company developed the recombinant form of theenzyme "to overcome supply constraints of the placentalproduct," LaVoie said, "we will be manufacturing and supplyingboth products" during the market introduction of therecombinant version. "There will be limited supplies of therecombinant product until Genzyme can complete theconstruction of its new production facility (which is in Allston),"LaVoie told BioWorld. That should be by late 1994 or early1995, she added.
And although over time Ceredase will be phased out, "it will besold as long as it's needed," said LaVoie.
Genzyme currently is providing Ceredase treatment for about800 patients worldwide at an annualized cost per patient ofabout $140,000. The company anticipates that the averagemaintenance cost of therapy will be below $60,000 per year.But the cost of that first-generation drug, which has orphandrug status, has been a source of irritation to some members ofCongress since at least 1991.
The latest volley was fired in early April by Rep. Fortney "Pete"Stark, D-Calif., who charged that Genzyme could realize awindfall profit of $214 million in one year on Ceredase sales.
The price of the second-generation product will be determinedat product introduction, LaVoie told BioWorld.
-- Jennifer Van Brunt Senior Editor
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