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The U.S. Food and Drug Administration has contacted Israel-based Protalix BioTherapeutics Inc. about the possibility of initiating a treatment protocol for use of its Phase 3 drug target for Gaucher’s disease. The drug has not yet been approved for use in the U.S. but would be used to blunt the effect that the Cerezyme shortage is having on patients, who have no other approved treatment options.
Cambridge-based Genzyme Corp. announced that supplies of its drug Cerezyme would be constrained for several weeks as the company works to get its Allston-based plant back on line. The plant was closed last month due to the discovery of a virus which does not infect humans but can alter the production of the drug.
Under current FDA regulations, a treatment protocol may be submitted for a drug that has not yet been approved for marketing but is the subject of clinical development for a serious or life threatening disease for which no comparable or satisfactory alternative drug or therapy is available. Protalix and the FDA are discussing the parameters of a proposed treatment protocol that would allow an increased number of patients with Gaucher disease to have access to the company’s lead product candidate, prGCD.
Gaucher disease is a rare and serious lysosomal storage disorder in humans with severe and debilitating symptoms. PrGCD is a proprietary plant-cell expressed recombinant form of glucocerebrosidase and is currently the subject of a Phase 3 clinical trial for the treatment of Gaucher disease.
Protalix’ (NYSE-Amex:PLX) stock jumped more than 14 percent on the news, to $5.15, up from the previous close of $4.51.
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