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FDA grants orphan drug designation to Toca 511 & Toca FC for glioblastoma
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The FDA granted orphan drug designation to Toca 511 & Toca FC for the treatment of patients with glioblastoma, according to a press release from the drug’s manufacturer.
Toca 511 & Toca FC (Tocagen Inc.) has the potential to kill tumor cells and activate the immune system selectively against the tumor. Toca 511 is a retroviral replicating vector that selectively delivers a gene for cytosine deaminase to the tumor. Toca FC is a novel formulation of an antifungal drug (flucytosine), which is converted into anticancer drug 5-fluorouracil within infected cancer cells.
Through a combination of mechanisms, immune activation locally in the tumor breaks the barrier of immune tolerance and may lead to durable tumor response.
In July, the FDA granted fast track designation to Toca 511 & Toca FC for the treatment of recurrent high-grade glioma, including glioblastoma and anaplastic astrocytoma.
"There's an extraordinary need for new treatment options for patients with this devastating disease," Harry Gruber, MD, CEO of Tocagen, said in a press release. "We believe FDA's granting of both orphan drug and fast track designations to Toca 511 & Toca FC will enable us to more efficiently advance our program, which we hope will ultimately offer physicians and patients a new option in the fight against brain cancer."
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.