The Food and Drug Administration has awarded orphan drug designation to BioSante Pharmaceuticals’ melanoma cancer vaccine.
The vaccine will be used to treat stage IIb-IV melanoma, says the company, in a statement. The Lincolnshire, Ill.–based BioSante estimates that 68,000 people were diagnosed with melanoma in 2010, and that 8,700 died. The FDA grants orphan status for products intended to treat fewer than 200,000 patients in the United States. According to the agency, makers of orphan products are eligible for tax credits and marketing incentives, but they still have to submit the same efficacy and safety data required of other products. Orphan status does not change the timetable for approval.
The BioSante vaccine is in early development at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center. It is a "GVAX" vaccine. According to a BioSante statement, it uses cell lines that are genetically modified to secrete granulocyte-macrophage colony–stimulating factor (GM-CSF). The cells are then irradiated to prevent further cell division. The GVAX model is also being used in BioSante products in development for pancreatic cancer, acute myeloid leukemia and chronic myeloid leukemia.