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