The U.S. Food and Drug Administration has granted Orphan Drug status to CGTG-102, a granulocyte-macrophage colony-stimulating factor-coding oncolytic adenovirus (Ad5/3-D24-GMCSF) for the treatment of soft tissue sarcoma. Although approximately 50 percent of these sarcomas are found in the early stage, for patients diagnosed at an advanced stage, overall survival is on average only about 13 months with the current standard of care.
CGTG-102 is based on a modified GM-CSF encoding adenovirus that selectively replicates in tumor cells, eventually killing them, and also releasing newly synthesized viruses that infect neighboring tumor cells. A Phase I study with the immunotherapy is planned to be completed this year—and Phase II studies are scheduled for 2014.
Prior to the Phase I study, 115 patients with chemotherapy-refractory solid tumors had been treated with CGTG-102 in an individualized treatment program regulated by the Finnish Medicines Agency as determined by the European Union Regulation on Advanced Therapy Medicinal Products, EC/1394/2007.
The Orphan Drug designation—designed to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
The drug also received the designation from the European Medicines Agency, which similarly provides incentives including fee reductions on future activities and the potential for 10 years of market exclusivity.