Protalix Biotherapeutics Inc. (AMEX:PLX) reports positive preliminary data from the first 15 patients in a worldwide switchover trial of its Gaucher disease treatment, taliglucerase alfa. The nine-month trial was conducted according to protocol cleared by the US Food and Drug Administration (FDA). The data indicate that patients can safely switch to taliglucerase alfa from imiglucerase (Cerezyme, made by Genzyme Inc (NYSE: DNA) unit Allston).
The preliminary data found no safety concerns from the switchover, and that drug efficacy was maintained. The switchover trial was originally designed to enroll 15 patients, but was expanded to 30 patients because of a shortage of enzyme replacement therapy for Gaucher patients. No more adults are being enrolled in the trial, but enrollment of children is still open.
Protalix president and CEO Dr. David Aviezer said, "We are pleased with the interim results of the switchover trial to taliglucerase alfa."
Protalix has begun sales of its drug in France, where it obtained a provisional permit in July, under a mechanism for still unauthorized drugs for which there is a public need. The FDA is still reviewing taliglucerase alfa under a Prescription Drug User Fee Action (PDUFA) date scheduled for February 25, 2011.
Gaucher diseases is a genetic disorder that affects enzymes necessary for intracellular digestion. Gaucher disease is caused by mutations or deficiencies in the gene encoding GCD, a lysosomal enzyme. Gaucher cells accumulate in the spleen, liver, lungs, bone marrow, and brain. Symptoms of Gaucher disease may include enlarged liver and spleen, abnormally low levels of red blood cells and platelets, and skeletal complications.
Protalix's share price rose 2.2% on the American Stock Exchange yesterday to $9.87, giving a market cap of $800 million.
Published by Globes [online], Israel business news - www.globes-online.com - on November 2, 2010
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