FDA approves Pfizer, Protalix drug for Gaucher disease

Agency grants approval to Elelyso

NEW YORK — The Food and Drug Administration has approved a new treatment for a rare genetic disorder that affects 10,000 people worldwide.

Pfizer and Protalix BioTherapeutics announced Wednesday the approval of Elelyso (taliglucerase alfa), an enzyme-replacement therapy for the long-term treatment of Type 1 Gaucher disease.

The companies said the drug was the first FDA-approved plant cell-based ERT for Gaucher disease and the first approved plant cell-expressed drug derived from Carmiel, Israel-based Protalix's ProCellEx manufacturing system, using genetically engineered carrot cells.

"The approval of Elelyso is important for patients who depend on available ERT to manage their Gaucher disease," Protalix president and CEO David Aviezer said. "At Protalix, our passion to develop Elelyso was strongly driven by our personal experience with family members and friends who have to live with this disease every day."


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