FDA gives orphan OK to Pluristem's delivery platform for Buerger's disease
Israeli biotech Pluristem has received orphan drug status from the FDA for its PLacental eXpanded (PLX) drug-delivery platform for the treatment of Buerger's disease, a rare condition affecting blood vessels in the extremities that sometimes can lead to amputation. Buerger's affects about 50,000 patients in the U.S. and Europe, but there are no established treatments for the disease, the company said in a release. It added that the market for treatment of the condition is about $2.5 billion.
"In anticipation of this designation, we have been working diligently in readying clinical sites, primarily in India, where there is a high prevalence of Buerger's," Pluristem CEO Zami Aberman said in a statement. "In addition, the inclusion of Buerger's completes our plan to make our PLX cells available for the entire spectrum of peripheral vascular disorders and allows us to benefit from the market exclusivity and other regulatory and financial advantages that accompany this designation."
Pluristem's PLX platform releases a cocktail of therapeutic proteins in response to a number of inflammatory diseases. In preclinical animal models, PLX cells were shown to be potentially effective in nerve pain and muscle damage--when administered locally--and in inflammatory bowel disease, multiple sclerosis and stroke--when administered systemically.