Pfizer and Protalix BioTherapeutics have announced FDA approval of Elelyso (taliglucerase alfa) for injection to treat type 1 Gaucher disease.
Elelyso, derived from ProCellEx, is an enzyme replacement therapy (ERT) for the long-term treatment of adults with type 1 Gaucher disease.
The plant cell-expressed drug is a form of the human lysosomal enzyme, glucocerebrosidase.
Protalix BioTherapeutics president and chief executive officer David Aviezer said the approval of Elelyso is important for patients who depend on available ERT to manage their Gaucher disease.
"We also believe that this great news is a recognition of our technology, which is a plant cell manufacturing system from Protalix. This technology is the production process behind ELELYSO and other Protalix product candidates," Aviezer added.
The approval is based on Protalix's clinical development program, which included a Phase III trial in which primary endpoint, as Elelyso significantly reduced spleen volume after nine months compared to baseline in both treatment groups, was achieved.
Secondary endpoints of liver volume, hemoglobin and platelet counts also showed improvement, in the study.
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.