Protalix BioTherapeutics, Inc. announced that, in connection with the New Drug Application (NDA) filed by the company in December 2009 for taliglucerase alfa for the treatment of Gaucher disease, the US Food and Drug Administration (FDA) has requested additional data regarding the Chemistry, Manufacturing and Controls (CMC) section of the NDA. No additional Clinical or Preclinical information was requested.
According to the company, the request focused primarily on validation of the manufacturing process in the company's upgraded manufacturing facility. A validation plan for the company's manufacturing process of taliglucerase alfa has already been established and reviewed by the FDA. The company is working diligently to provide the requested data to the FDA and anticipates submitting the requested data during the second quarter of 2010.
Based on the FDA's request as part of its rolling review of the NDA, the Prescription Drug User Fee Act (PDUFA) action date for taliglucerase alfa is expected to be issued following submission of the additional requested CMC data.
Taliglucerase alfa will continue being provided to Gaucher patients in the United States under an Expanded Access protocol, as well as to patients in the European Union, Israel and other countries under Named Patient provisions.
Gaucher disease, an inherited condition, is the most prevalent lysosomal storage disorder, with an incidence of about 1 in 20,000 live births. People with Gaucher disease do not have enough of an enzyme, beta-glucosidase (glucocerebrosidase), that breaks down a certain type of fat molecule. As a result, lipid engorged cells (called Gaucher cells) amass in different parts of the body, primarily the spleen, liver and bone marrow. Accumulation of Gaucher cells may cause spleen and liver enlargement, anemia, excessive bleeding and bruising, bone disease and a number of other signs and symptoms.
Protalix is a biopharmaceutical company focused on the development and commercialization of proprietary recombinant therapeutic proteins expressed through its proprietary plant cell based expression system.