Protalix, Pfizer Report FDA Approval of Plant-Derived Gaucher Disease ERT Elelyso

Protalix BioTherapeutics and partner Pfizer reported FDA approval of Elelyso™ (taliglucerase alfa), a plant cell-produced glucocerebrosidase enzyme replacement therapy (ERT) for the long-term treatment of adult patients with type 1 Gaucher disease. Protalix says that as well as being the first FDA-approved plant-cell based ERT for Gaucher disease, Elelyso is also the first approved plant cell-expressed drug generated through its ProCellEx® manufacturing platform, which uses genetically engineered carrot cells.

FDA clearance of Elelyso was based on data from clinical trials which demonstrated the noninferiority of the drug compared with the approved Gaucher disease ERT imiglucerase. Another clinical study confirmed that treating previously ERT-naive patients with Elelyso led to significant reductions in spleen and liver volumes, and improved hemoglobin and platelet counts.

Protalix and Pfizer inked their worldwide development and commercialization deal for Elelyso in 2009. Under terms of the deal Pfizer negotiated exclusive commercialization rights to the ERT worldwide, except for Israel, where Protalix retains exclusive commercialization rights. Marketing applications for taliglucerase alfa have in addition been submitted in Europe, Israel, Brazil, and Australia. Regulatory submissions in other countries are in progress.

Israel-based Protalix is exploiting its ProCellEx production system to develop novel and biosimilar protein-based therapeutics both in house and through partnerships with the biopharma industry. Its clinical pipeline includes PRX-105, a Phase I-stage PEGylated recombinant human acetylcholinestrase (AChE), which is in development primarily for biodefense applications as a therapeutic and prophylactic countermeasure for nerve agents attack. Protalix has in addition demonstrated that PRX-105 may have applications in the treatment of Parkinson disease.

The firm’s preclinical pipeline includes potential candidates for the treatment of Fabry disese and autoimmune diseases, as well as an orally administered glucocerebrosidase.