FDA granted Orphan Drug Designation to Toca 511 & Toca FC, an investigational immunotherapy treatment for glioblastoma developed by Tocagen.
The agency recently granted the drug Fast Track designation for the treatment of recurrent high-grade glioma, which includes glioblastoma and anaplastic astrocytoma. According to Tocagen, the drug is planned to move into a clinical trial later this year.
Toca 511 & Toca FC is an investigational treatment that is designed to program cancer cells to convert the prodrug 5-FC into the anticancer drug 5-FU, killing tumor cells and leading to activation of the immune system via a combination of mechanisms.
Toca 511 is a retroviral replicating vector that selectively delivers a gene for the enzyme cytosine deaminase to the tumor. Patients then take oral cycles of Toca FC, a novel formulation of an antifungal drug, which is converted within infected cancer cells into the FDA-approved anticancer drug, 5-fluorouracil. Immune activation locally in the tumor occurs through a combination of mechanisms that together break the barrier of immune tolerance and may lead to durable tumor response, according to Tocagen.
