FDA Issues Complete Response Letter for Taliglucerase Alfa New Drug Application

Protalix BioTherapeutics, Inc. PLX, announced earlier today that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter regarding the Company's New Drug Application (NDA) for taliglucerase alfa for the treatment of Gaucher disease. Taliglucerase alfa is a plant-cell expressed form of glucocerebrosidase. A CRL is issued by the FDA's Center for Drug Evaluation and Research when the review of a file is completed and questions remain that preclude the approval of the NDA in its current form. The main questions raised by the FDA regarding the NDA relate to clinical and chemistry, manufacturing and controls (CMC). In the clinical section, the FDA requested additional data from the Company's switchover trial and long-term extension trial. At the time the NDA was submitted, full data from these trials was not available. In the CMC section, the FDA requested information regarding testing specifications and assay validation. "While we are disappointed by the receipt of the Complete Response Letter, we appreciate the FDA's efforts to complete the review of our NDA. We noted that the FDA did not request additional clinical studies. Moreover, the FDA inspected our manufacturing facilities finding them acceptable. FDA also did not identify any issues in its audit of our clinical sites," said Dr. David Aviezer, the Company's President and Chief Executive Officer. "Protalix will work with the FDA to determine next steps."