The extended treatment protocol allows Protelix to significantly increase the number of patients treated with the drug in the US and other countries while continuing the research as part of a phase III trial
- Protlix Biotherapeutics announced that the US Food and Drug Administration (FDA) approved the extended treatment protocol for the use of the prGCD drug in patients with Gaucher's disease. Gaucher's disease is a rare and serious disorder that manifests itself in severe and debilitating symptoms and is caused by a lack of the enzyme glucocerebrosidase to break down fats. prGCD, the leading product of Protelix, is based on a technology developed by the company for the production of recombinant proteins on a plant cell basis. prGCD is the subject of research in a phase III clinical trial examining the treatment of Gaucher's disease.
Protelix was asked by the US Food and Drug Administration to submit an extended treatment protocol due to the shortage of the existing drug manufactured by the Genzyme company. The expanded treatment protocol allows Protelix to significantly increase the number of patients treated with the drug in the US and other countries while continuing the research as part of a phase III trial. Before approving the protocol, the FDA reviewed a recent report that described the current status of all the prGCD drug trials.
The treatment protocol is multicenter, randomized, designed to test the safety of the prGCD and allows doctors to treat patients with Gauche with the drug during the expected period of enzyme deficiency and beyond. The patients will be able to continue to be treated with prGCD until the drug is commercially available. The company will provide the drug free of charge to patients who will be recruited as part of the protocol.
Dr. David Aviezer - President and CEO of Protelix: "We appreciate the cooperation given by the US Food and Drug Administration in preparing the treatment protocol and we work continuously and closely with doctors and patient organizations in order to enable treatment with the drug. We expect the results of the phase three trial to be published in October and plan to submit the registration file to the authorities by the end of 2009."
Protelix produces the recombinant human glucocerebrosidase protein in plant cells. This protein is needed for Gaucher patients and its production in plants on an industrial scale is a tremendous achievement for the Israeli biotechnology industry.
