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news.Tocagen, a clinical-stage immuno-oncology company, announced that the US 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.
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As previously announced, the investigational drug was granted FDA Fast Track designation for the treatment of recurrent high-grade glioma, which includes glioblastoma and anaplastic astrocytoma, and is poised to move into a pivotal clinical trial later this year.
Glioblastoma is the most common form of primary brain cancer, with over 10,000 people diagnosed every year in the United States. The disease is extremely aggressive; newly diagnosed patients have a five-year survival rate of less than 5 percent, and the tumor typically recurs following standard-of-care treatment.
Median survival following recurrence is approximately eight months. 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.
"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."
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.
Orphan drug designation qualifies the drug developer for various development incentives, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee for the condition for which the drug was designated.
Orphan designation does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.