DOW JONES NEWSWIRES
Protalix BioTherapeutics (PLX) reported its Gaucher's disease treatment met the primary goal of a late-stage trial, setting the stage for Uplyso's approval by the Food and Drug Administration.
The FDA in late August gave Protalix fast-track status, speeding up review of the Gaucher's treatment amid an expected shortage of Genzyme Corp.'s (GENZ) Cerezyme. It had to shut production in June after a virus was found.
The agency in August also cleared Uplyso for patients with the rare, inherited disorder - in which an enzyme problem results in harmful quantities of certain fats, especially within the bone marrow, spleen and liver.
Although the treatments from British drug maker Shire Ltd. (SHPGY, SHP.LN) and Israeli-based Protalix aren't approved for sale in the U.S., the companies were permitted to offer their drugs to U.S. residents under special treatment protocols approved by the FDA.
Protalix said Thursday that Uplyso not only met its primary goal but that secondary endpoints showed "statistically significant improvement" as well.
Shares closed Wednesday at $8.88 and were inactive premarket. The stock is up nearly five-fold this year.
-By Kevin Kingsbury; Dow Jones Newswires; 212-416-2354; kevin.kingsbury@dowjones.com