NEW YORK The Food and Drug Administration has approved a treatment protocol for a drug to treat a rare but potentially deadly genetic disease.
Protalix BioTherapeutics announced the FDA’s approval of its treatment protocol plant-cell expressed recombinant form of glucocerebrosidase for the treatment of Gaucher disease, currently in phase 3 studies.
The protocol is a multicenter, open-label trial that will allow healthcare providers to use prGCD to treat patients with Gaucher disease during an expected shortage of Genzyme’s Gaucher treatment, Cerezyme.
“We appreciate the guidance and vote of confidence provided by the FDA in establishing a treatment protocol for prGCD and are working closely with physicians and patient advocacy groups to allow Gaucher disease patients to gain access to our drug,” Protalix president and CEO David Aviezer said in a statement. “We expect to continue our phase 3 pivotal study next month and are looking forward to announcing top-line results from this study in October.”
According to the National Gaucher Foundation, Gaucher disease occurs in about one-in-20,000 live births and is particularly prevalent in people of Ashkenazi Jewish descent. Patients with the disease have a genetic lack of ability to produce glucocerebrosidase, or GCD, in their bodies, resulting in the buildup of fatty tissue that can lead to enlargement of internal organs and death.