Elelyso: a new therapeutic option for patients with Gaucher disease

The US Food and Drug Administration recently approved Protalix BioTherapeutic’s (NYSE-AMEX: OLX) Elelyso (taliglucerase alfa), a new enzyme replacement therapy (ERT) that provides patients with Gaucher disease, which will be marketed by global drugs behemoth Pfizer (NYSE: PFE; The Pharma Letter May 2).

Elelyso is an alternative therapeutic option to Genzyme’s Cerezyme (imiglucerase), the current market leader and standard of care in Gaucher disease, and Shire’s Vpriv (velaglucerase alfa). Elelyso is the first ERT that is manufactured using a plant cell-based process, and the drug’s lower pricing as compared to both Cerezyme and Vpriv has been attributed to reduced costs associated with the plant cell production platform, comments pharma industry experts at GlobalData.

Elelyso was developed and is now being promoted through a partnership between Protalix and Pfizer. The FDA and the European Medicines Agency both granted Elelyso orphan drug status. The FDA also initially granted Elelyso fast-track development status, and a pivotal Phase III clinical trial, which served as the basis for the NDA submission, was completed in September 2009. Elelyso and Vpriv were both approved for early access programs for patients requiring ERT during the global shortage of Cerezyme between 2009 and 2010, which resulted from a viral contamination incident at Genzyme’s Allston Landing facility in Massachusetts.