CORRECT: FDA Approves Protalix, Pfizer Drug To Treat Gaucher Disease
May 01 2012 - 5:37PM
Dow Jones News
The Food and Drug Administration Tuesday approved a new
treatment developed by Protalix Bio Therapeutics Inc. (PLX, PLX.TV)
for adults with a type of Gaucher disease, a rare genetic
disorder.
The treatment, taliglucerase alfa, will be sold in the U.S.
under the brand name Elelyso by Pfizer Inc. (PFE).
Pfizer also will sell the drug in most places outside the U.S.
The drug, which is the first plant-derived cell-based enzyme
replacement, replaces an enzyme called glucocerebrosidase in people
with type 1 Gaucher disease. Elelyso is produced using genetically
engineered carrot cells.
People with Gaucher disease lack glucocerebrosidase, which
causes an accumulation of a fatty substance inside cells. The fatty
accumulation can lead to enlargement and malfunctioning of the
liver, spleen, bone marrow and occasionally, the lung, kidney, and
intestine. About 6,000 people in the U.S. have type 1 Gaucher
disease according to FDA.
Pfizer and Protalix have already been making Elelyso available
to some Gaucher disease patients as part of an early access program
because of manufacturing issues at Genzyme, a unit of Sanofi (SNY)
which makes another glucocerebrosidase-enzyme replacement product,
Cerezyme. Earlier this year Genzyme said the manufacturing problems
had been fixed and that the company is able to make enough of the
product to supply current patients in the U.S.
Shire PLC (SHPGY, SHP.LN) which also makes a Gaucher disease
treatment, Vpriv, has said inventories of the product will be
"below target levels" until the FDA signs off on a second
manufacturing plant in the U.S. However, the company also said it
has the capacity to meet anticipated demand for Vpriv.
Officials from Pfizer and Protalix said the companies have
already built enough inventory of Elelyso to supply to patients for
at least 24 months.
Elelyso is an infusion that is administered by health-care
professionals every other week.
-By Jennifer Corbett Dooren, Dow Jones Newswires; 202-862-9294;
jennifer.corbett@dowjones.com