The U.S. Food and Drug Administration has granted Orphan Drug status to Toca 511 & Toca FC for the treatment of patients with glioblastoma.
Toca 511 & Toca FC, made by Tocagen Inc., is an investigational treatment designed to program cancer cells to covert the prodrug 5-FC into the fluorouracil, killing tumor cells and leading to activation of the immune system via a combination of mechanisms.
Toca 511 & Toca FC had also been granted the FDA's Fast Track designation for the treatment of patients with high-grade glioma, including glioblastoma and anaplastic astrocytoma.
The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
The treatment will be evaluated in a Phase II/III study in patients with recurrent glioblastoma or anaplastic astrocytoma beginning later this year, according to a news release from the company. A study evaluating Toca 511 & Toca FC in five additional metastatic solid tumor indications is planned for next year.