Protalix BioTherapeutics of Carmiel, Israel has successfully completed a pivotal Phase III clinical trial for its lead product candidate, UPLYSO, to be used in enzyme replacement therapy for Gaucher Disease.

Rare and serious, Gaucher Disease is a lysosomal storage disorder with severe and debilitating symptoms. It results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents.

The trial, comprising 31 patients enrolled at 11 centers in Israel, Europe, North America, South America and South Africa, met its primary endpoint, which was stipulated in the Special Protocol Assessment agreed on by the biopharmaceutical company and the US Food and Drug Administration (FDA) prior to commencement of the trial.

“The results of this pivotal trial suggest that UPLYSO has the potential to become an attractive treatment alternative for Gaucher Disease patients,” said Professor Ari Zimran, Director of the Gaucher Clinic at Shaare Zedek Medical Center in Jerusalem and Coordinating Investigator of the UPLYSO trial.

In July, the FDA asked Protalix to submit a treatment protocol for its lead product.

Protalix is currently making UPLYSO available to Gaucher disease patients in the US and other countries under an Expanded Access Program approved by the FDA.