The medication also received Fast Track status in July..
The VX15 antibody is currently being evaluated in a Phase II clinical trial. It was designed to block semaphorin 4D (SEMA4D) activation of microglia and astrocytes, which are the central nervous system's inflammatory cells. These cells are believed to be a mechanism in HD, multiple sclerosis (MS) and other disorders.
“We are pleased to receive Orphan Drug Designation from the FDA for VX15 as a potential treatment for Huntington’s disease," Vaccinex CEO Maurice Zauderer said. "SIGNAL is an important proof of concept study for the role of inflammation and, in particular, SEMA4D in neurodegenerative processes. We hope that this study will contribute to treatment of HD, a serious disease for which there are, as yet, no approved disease-modifying therapies.”
Orphan drug designation encourages development of drugs for rare diseases and condition that affect fewer than 200,000 people in the U.S. In some cases, the disease may affect more than 200,000 patients but developing a treatment would not be economically feasible. The designation also provides seven years of market exclusivity, tax credits and FDA user fee waivers.