Protalix Biotherapeutics has had a tough time landing its first product on the market. Since the small Israeli company filed for approval for Uplyso (taliglucerase alfa) with the US Food and Drug Administration (FDA) two years ago, the agency has twice requested additional data about the manufacturing and long-term clinical benefits of the drug, a recombinant enzyme developed in partnership with the US pharma giant Pfizer to replace the defective protein found in people with Gaucher's disease. After responding to the agency's concerns, Protalix had expected a final decision on Uplyso by February. But, last month, the company announced that that date had been pushed back to the start of May as the FDA mulls over more late-breaking clinical information.
The clock is ticking. The market for Gaucher's drugs is relatively small to begin with, as fewer than 10,000 people, mostly of Ashkenazi Jewish descent, are affected by the lysosomal storage disorder worldwide. Plus, it's become an increasingly crowded space: Cerezyme (imiglucerase), a similar enzyme replacement therapy from the US biotech company Genzyme, currently dominates the market, with global sales of around $720 million in 2010, although shortages have recently plagued its distribution; and, in February 2010, another player—Ireland's Shire—entered the field with VPRIV (velaglucerase alfa), which netted $143 million in its first year alone.
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