Protalix BioTherapeutics, Inc. was recently approached by the FDA and asked to consider submitting a treatment protocol for the use of prGCD in patients with Gaucher disease in order to address an expected shortage of the drug Cerezyme®, a mammalian cell expressed version of glucocerebrosidase (GCD) and the only enzyme replacement therapy currently approved for Gaucher disease. Gaucher disease is a rare and serious lysosomal storage disorder in humans with severe and debilitating symptoms. prGCD, the Company’s lead product candidate, is a proprietary plant-cell expressed recombinant form of glucocerebrosidase and is currently the subject of a Phase III clinical trial for the treatment of Gaucher disease. The FDA indicated to the company that it believes the company’s development program for prGCD satisfies the regulatory criteria required to supply prGCD for expanded access to patients under a treatment protocol. 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. The Company and the FDA are discussing the parameters of a proposed treatment protocol which would allow an increased number of patients with Gaucher disease to have access to prGCD. The Company expects to submit a treatment protocol to the FDA for its review as a supplement to the Company’s current Investigational New Drug (IND) application for prGCD.
