Tocagen’s Double Action Glioblastoma Treatment Receives FDA Orphan Drug Designation

Tocagen’s Double Action Glioblastoma Treatment Receives FDA Orphan Drug Designation

Clinical stage immuno-oncology company, Tocagen, Inc., announced the US Food and Drug Administration has granted its primary immuno-oncology candidate orphan drug designation as a promising and much-needed treatment of glioblastoma, the most common form of primary brain cancer. Every year, over 10,000 people are diagnosed with glioblastoma in the United States. The new designation brings the company’s Toca 511 & Toca FC closer to helping patients suffering with this type of tumor. Tocagen is preparing to proceed with a pivotal clinical trials later this year.

Glioblastoma is known to be extremely aggressive, with newly diagnosed patients expecting a mere five-year survival rate of less than 5 percent, along with a high likelihood of tumor recurrence despite completion of standard treatment. Once the tumor recurs, the average survival is only 8 months.

Toca 511 is a retroviral replicating vector (RRV) that selectively delivers a gene for the enzyme cytosine deaminase into 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 (5 FU). Toca 511 & Toca FC work by programming cancer cells to convert the prodrug 5-FC into the anticancer drug 5-FU, effectively causing tumor cell death and stimulating the immune system through a combination of mechanisms.

“There’s an extraordinary need for new treatment options for patients with this devastating disease,” said Harry Gruber, M.D., chief executive officer of Tocagen. “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.”


ImmunoCellular Therapeutics, Ltd., announced it has come to an agreement with the US Food and Drug Administration (FDA) on a Special Protocol Assignment (SPA) for the Phase III registrational study of its investigational immunotherapy, ICT-107, indicated for patients with glioblastoma.

ICT-107 is a dendritic cell-based immunotherapy targeting multiple tumor-associated antigens on glioblastoma stem cells. The trial will be a randomized, double-blind, placebo-controlled, and will aim to enroll around 400 HLA-A2 positive patients. The study will be conducted across 120 sites in the US, Canada, and the European Union.

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