Tocagen receives orphan drug designation for glioblastoma treatment

Tocagen, Inc. announced Tuesday that the U.S. Food and Drug Administration has granted orphan drug designation to the company's lead immuno-oncology product candidate, Toca 511 & Toca FC, for the treatment of glioblastoma.

"There's an extraordinary need for new treatment options for patients with this devastating disease," Harry Gruber, CEO of Tocagen, said. "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."

Glioblastoma is the most common form of primary brain cancer. More than 10,000 people are diagnosed with glioblastoma every year in the United States. The disease is very aggressive and newly diagnosed patients have a five-year survival rate of less than 5 percent. The tumor typically recurs following standard-of-care treatment.

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.

The FDA's orphan drug designation is intended to encourage development of treatments for diseases that affect fewer than 200,000 individuals in the United States.