Protalix Gets FDA Approval

Pfizer Inc.(NYSE:PFE) and Protalix BioTherapeutics, Inc. (NYSE-AMEX:PLX, TASE:PLX) announced that the United States (U.S.) Food and Drug Administration (FDA) approved ELELYSO(TM) (taliglucerase alfa) for injection, an enzyme replacement therapy (ERT) for the long-term treatment of adults with a confirmed diagnosis of type 1 Gaucher disease.

ELELYSO is the first FDA-approved plant cell-based ERT for Gaucher disease. It is also the first approved plant cell-expressed drug that is derived from ProCellEx(R), Protalix’s proprietary manufacturing system, using genetically engineered carrot cells. ELELYSO(TM) is a form of the human lysosomal enzyme, glucocerebrosidase, used to treat Gaucher disease.

Supply disruptions of approved ERTs have been affecting those living with Gaucher disease since 2009 in multiple countries including the U.S. To help minimize the possibility of supply disruptions, Pfizer is launching the “Supply Continuity Program,” which will endeavor to maintain a continuously restocked 24 months of supply at various stages of production for U.S. patients prescribed ELELYSO.

The approval of ELELYSO(TM) (taliglucerase alfa) is based on the review of Protalix’s clinical development program. In a study of 31 adult patients with Type 1 Gaucher disease naïve to enzyme replacement therapy, the safety and efficacy of ELELYSO was assessed. The trial was a nine-month, multi-center, double blind, randomized study in patients with Gaucher disease-related enlarged spleens and thrombocytopenia. Patients were randomized to receive ELELYSO at a dose of either 30 Units/kg (n=15) or 60 Units/kg (n=16). Data showed the pivotal phase III clinical trial achieved its primary endpoint as ELELYSO significantly reduced spleen volume after nine months compared to baseline in both treatment groups. Secondary endpoints of liver volume, hemoglobin and platelet counts also showed improvement. Twenty-six patients continued to be treated with ELELYSO in an extension of this study in a blinded manner for a total treatment duration of 24 months. The data demonstrated continued improvement in the clinical parameters. In a study of 25 patients with Type 1 Gaucher disease who were switched from imiglucerase to ELELYSO, the safety and efficacy of ELELYSO was assessed. The trial was a nine-month, multi-center, open-label, single arm study in patients who had been receiving treatment with imiglucerase at doses ranging from 11 Units/kg to 60 Units/kg for a minimum of 2 years. Imiglucerase therapy was stopped, and treatment with ELELYSO was administered every other week at the same number of units as each patient’s previous imiglucerase dose. Organ volumes and hematologic values remained stable on average through nine months of ELELYSO treatment.

Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell based expression system, ProCellEx(R). Protalix’s unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix’s first approved product manufactured by ProCellEx, ELELYSO(TM) (taliglucerase alfa), an enzyme replacement therapy for the treatment of Gaucher disease, was approved for marketing by the U.S. Food and Drug Administration on May 1, 2012. Additional marketing applications for taliglucerase alfa have been filed in other countries.