October 29, 2002
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) will convene on January 13, 2003 to consider Genzyme’s (NASDAQ:
GENZ) Fabrazyme, and will consider Transkaryotic’s (NASDAQ:
TKTX) Replagal the next day. Both enzyme replacement therapies are designed to treat Fabry disease, both have orphan drug status in the U.S., and both are marketed in Europe, sharing ten years’ market exclusivity.
Available clinical trial data suggests that both drugs can have a significant impact on Fabry disease course, but regulatory concerns and European post-marketing anecdotes have tipped the scales for first FDA approval in Fabrazyme favor. Although the U.S. Orphan Drug Act typically grants approval for only one orphan drug per indication with seven years of market exclusivity, precedent certainly exists for shared or shortened duration of such status.
The EMDAC will report its findings to the FDA, which is expected to issue a final marketing status decision during the first half of 2003. Our modeling estimates the probability of Fabrazyme approval at 85-90% and the probability of Replagal approval at 75-85%.
The EMDAC will also meet on January 15 to consider BioMarin (NASDAQ:
BMRN) and Genzyme’s Aldurazyme (laronidase), an enzyme replacement therapy for patients with mucopolysaccharidosis I (MPS I). Aldurazyme is also in regulatory review at the European Agency for the Evaluation of Medicinal Products (EMEA), and the companies expect a response from the European regulators in the first half of 2003. Our modeling estimates the probability of Aldurazyme approval at 90-95%.
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