FDA Gives Investigational Drug Orphan Status
05/13/03 Portland, Ore.
Multiple sclerosis drug developed by Virogenomics and OHSU/VA scientists
The FDA's orphan drug program is intended to encourage research, development and regulatory approval of products for diseases that affect fewer than 200,000 patients in the United States. In addition to potential market exclusivity, orphan drug status provides tax incentives for up to 50 percent of a company's investment in U.S. clinical research, partial funding to support clinical trials, study design assistance and waivers from FDA user fees.
"Orphan drug status is a significant step in the commercialization of a discovery made by OHSU scientists," said Todd Sherer, Ph.D., director of OHSU Technology and Research Collaborations, which spun off the drug technology to Virogenomics. "This will help bring VG1000 to market."
MS is caused when T cells that normally lead the attack against foreign invaders of the body instead target nerves in the spinal cord and brain, creating lesions in myelin, the protective sheath around nerve cells.
In animal models of MS, treatment with VG1000 restored normal neurological function. The drug targets T cells that cause inflammation and myelin damage in the brain and spinal cord.
"VG1000 has been shown to be safe and efficacious in three different animal models of MS," said Arthur Vandenbark, Ph.D., co-developer of VG1000; research professor of neurology and professor of molecular microbiology and immunology in the OHSU School of Medicine; and research career scientist at the Portland Veterans Affairs Medical Center. "Pre-clinical animal studies are being completed in anticipation of submitting an Investigational New Drug application to the FDA."
No effective prevention or cure for MS exists today. Current treatments are aimed at controlling symptoms and maintaining function to give maximum quality of life. The clinical effectiveness of these drugs is marginal and patients often have significant side effects.
MS usually strikes young adults aged 20 to 40. Women are twice as likely to be affected than men. The progress, severity and specific symptoms of the disease cannot be predicted; symptoms may range from tingling and numbness to paralysis and blindness.
Co-developers of VG1000 include Gregory G. Burrows, Ph.D., research associate professor of neurology, and research assistant professor of biochemistry and molecular biology in the OHSU School of Medicine; and Halina Offner, Dr. Med., research professor of neurology in the OHSU School of Medicine.
"What this team of scientists has done in creating VG1000 is truly remarkable," said Dennis Bourdette, M.D., interim chairman of neurology in the OHSU School of Medicine and director of the OHSU MS Center of Oregon. "They have genetically engineered a protein that can inactivate the T cells that we believe cause MS."
Virogenomics, an OHSU spin off, was formed in 2001, as a genomics-enabled drug discovery company developing two innovative platforms. The autoimmune platform is based on a proprietary class of compounds that can be tailored to specifically target a wide range of autoimmune diseases. The drug discovery platform uses a unique virus-induced process to identify patterns of gene expression for a given disease, and to study those patterns as a means of discovering novel gene targets and compounds directed against those targets.
Under the license agreement, OHSU is a shareholder of Virogenomics. The university also receives funding from Virogenomics to support the research, and is entitled to royalties and a share of sublicense fees on any products commercialized by Virogenomics as a result of these technologies.