Pfizer's Elelyso approved in USA for Gaucher disease; Celebrex settlement

The US Food and Drug Administration yesterday approved Elelyso (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, to be marketed by global pharma giant Pfizer (NYSE: PFE) under license from Israeli firm Protalix BioTherapeutics (NYSE-AMEX: PLX).

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, Protalix' proprietary manufacturing system, using genetically engineered carrot cells. Elelyso is a form of the human lysosomal enzyme, glucocerebrosidase, used to treat Gaucher disease.

Last year the FDA delayed clearing the drug, issuing a complete response letter, to which Protalix responded last summer raising questions 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. (The Pharma Letter February 12).