The U.S. Food and Drug Administration has granted a fast-track review to Protalix BioTherapeutics‘ experimental treatment for Gaucher disease. Fast-track designation allows the agency to review late-stage data as it becomes available, instead of waiting for complete Phase III data to be submitted. The designation is used to expedite the drug review process so patients with serious or life-threatening diseases can get access to experimental drugs sooner. Protalix plans to submit clinical trial data in October.
Individuals with Gaucher disease lack an enzyme that helps the body metabolize fats, causing fatty substances to accumulate in vital organs and bone marrow. Common symptoms of Gaucher disease include enlargement of the liver or spleen, anemia, and osteoporosis. The disease affects an estimated 8,000 to 10,000 people worldwide.
Genzyme‘s Cerezyme is the only approved treatment on the market at this time. However, the discovery of a virus at the company’s production facility in June has forced Genzyme to throw out most of the raw materials required to make the drug. Patients with Gaucher’s disease are now facing shortages and rationing of Cerezyme–part of the reason for the fast-track designation granted to Protalix.
Only a few other companies are developing treatments for Gaucher disease: Amicus Therapeutics completed a Phase II clinical trial for their Gaucher drug AT2101 in March 2008. Shire Therapeutics has reported positive results in the first of three Phase III trials for their enzyme replacement drug. Like Protalix, Shire has also received fast-track designation for their Gaucher treatment.