DNAtrix, an experts in oncolytic virus development, announced that the US Food and Drug Administration (US FDA) has granted Orphan Drug Designation for DNX-2401, a conditionally-replicative oncolytic adenovirus for malignant glioma. Glioma is the most common form of primary brain cancer, the treatment of which remains a significant unmet medical need. Under the designation, companies are provided with development and commercial incentives for designated compounds. The company’s DNX-2401 programme has already been granted fast track status.
The FDA’s orphan drug programme designates a special status to drugs and biologics intended to treat, diagnose, or prevent diseases and disorders that affect fewer than 200,000 people in the US. It can be easier to gain marketing approval for drugs with orphan status, and orphan drugs typically also enjoy extended marketing exclusivity periods.
“We are pleased to have been granted Orphan Drug designation for our lead glioma oncolytic virus therapy,” said Dr. Frank Tufaro, president and chief executive officer of DNAtrix. “Results from our clinical trials with DNX-2401 continue to indicate that the drug may be an important treatment option for the disease.”
Oncolytic virus therapy is based on the concept of using live viruses to selectively infect and replicate in cancer cells, with minimal destruction of normal tissue. Virus replication amplifies the input dose of the oncolytic virus and helps spread the agent to adjacent tumor cells. Moreover, there is evidence for a long-lasting anti-glioma immune effect that can lead to durable tumor destruction and long-term survival in some patients. DNX-2401, which has been engineered to be highly potent and selective for killing tumors, is currently being evaluated in clinical studies in the United States and Europe.