January 14, 2003
In the first of two meetings on drugs being reviewed for treating Fabry disease, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) met yesterday to consider Genzyme’s (NASDAQ:
GENZ) Fabrazyme. The committee will meet today to consider Transkaryotic’s (TKT, NASDAQ:
TKTX) Replagal. The two drugs, both already approved in Europe, have been vying for orphan drug status in the U.S. Fabrazyme endorsements by EMDAC as well as negative comments from an FDA staff report concerning Replagal tip the balance further in favor of Fabrazyme being the sole recipient of orphan drug status for Fabry disease in the U.S.
The EMDAC made the following conclusions about Fabrazyme:
- Phase III data demonstrating that Fabrazyme sufficiently clears accumulated globotriaoslyceramide from the blood vessels of the kidney is important as such clearance “is an appropriate surrogate marker for purposes of accelerated approval.” (committee vote: 14-1)
- Data on antibody formation to Fabrazyme do not suggest that the agent’s effects would diminish over time. The degree to which antibodies form against the product, and its clinical significance, should be evaluated in post-marketing investigations.
Fabrazyme’s risk/benefit profile is reassuring.
- Genzyme should persist in efforts designed to complete an ongoing placebo-controlled, phase IV study as currently designed.
- Fabrazyme’s Prescription Drug User Fee Act (PDUFA) date occurs at the end of April, prior to which FDA action is anticipated. The FDA has not asked its committee to vote on whether to approve either Replagal or Fabrazyme, but the recommendations of the EMDAC reduce the probability that the FDA might have concerns about Fabrazyme that are similar to the concerns the FDA has expressed about Replagal.
The EMDAC is unlikely to have as favorable an opinion of Replagal. An FDA staff report posted yesterday reiterated Replagal’s failure to meet specific goals relating to endpoints of pain and kidney function. Moreover, the report made clearer that regulatory staff also consider the heart-related endpoints unmet.
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