The Protalix BioTherapeutics biopharmaceutical company has announced that it was approached by the US Food and Drug Administration (FDA) and asked to consider submitting a treatment protocol for the use of its product prGCD, in patients with Gaucher disease, in order to address an expected shortage of the drug Cerezyme, which is the only enzyme replacement therapy currently approved for Gaucher disease.
Gaucher disease is a rare and serious lysosomal storage disorder with severe and debilitating symptoms. prGCD, Protalix’s lead product candidate, is currently the subject of a Phase III clinical trial for the treatment of Gaucher disease.
Protalix and the FDA are discussing the parameters of a proposed treatment protocol which would allow an increased number of patients with Gaucher disease to have access to prGCD.
Under current FDA regulations, a treatment protocol may be submitted for a drug that has not yet been approved for marketing but is the subject of clinical development for a serious or life threatening disease for which no comparable or satisfactory alternative drug or therapy is available.