Hit with a sudden shortage of drugs for Gaucher's disease, Protalix announced this morning that the FDA had approached the company to start providing its experimental therapy prGCD to patients under an expanded access program. And the news immediately fueled a 22 percent hike in the company's share price.
The developer says it is now discussing a treatment protocol with regulators which would expand the use of the therapy, a plant-cell expressed recombinant form of glucocerebrosidase now in a late-stage trial. Gaucher's disease is an inherited enzyme disorder which causes the harmful accumulation of certain fats.
The shortage was triggered by Genzyme's move in mid-June to halt production of Cerezyme after a virus had contaminated its Boston production facility. Cerezyme is Genzyme's biggest revenue generator, earning $1.2 billion last year.