FDA Expands Orphan Drug Designation for SurVaxM, Immunotherapy Developed at Roswell Park

Michael Ciesielski, PhD

The U.S. Food and Drug Administration (FDA) has expanded orphan drug designation for SurVaxM, a brain cancer immunotherapy born in the labs of Roswell Park Comprehensive Cancer Center. The supplemental designation awarded to MimiVax Inc. now applies to SurVaxM as treatment for not just adult glioblastoma but a broader category of cancerous brain tumors: any malignant glioma in children or adults.

Orphan status is a designation awarded by the FDA to encourage research and development of drugs that have shown promise in treating, preventing or diagnosing rare diseases. SurVaxM is a synthetic cancer vaccine that stimulates the immune system to attack tumor cells that carry a protein called survivin.

MimiVax first received orphan drug status for SurVaxM in August 2017, for treatment of adult glioblastoma. The supplemental designation greatly expands the potential for SurVaxM’s use in other forms of pediatric and adult malignant gliomas. Gliomas are the most common type of cancer originating in the brain. 

“Receiving orphan drug designations for SurVaxM emphasizes the critical demand for novel and enhanced therapeutic options for people living with malignant gliomas,” says Michael Ciesielski, PhD, Assistant Professor of Neurosurgery at Roswell Park and CEO of MimiVax. “We hope that this designation may help to advance SurVaxM’s application for important indications beyond adult glioblastoma, including for pediatric brain cancers.”

SurVaxM was invented at Roswell Park by Dr. Ciesielski and Robert Fenstermaker, MD, Roswell Park Chair of Neurosurgery. “We are greatly encouraged by the new enhanced designation as we begin to explore SurVaxM’s potential effectiveness against brain cancers other than glioblastoma,” says Dr. Fenstermaker, who is also Professor of Neurosurgery and Oncology and Director of Neuro-Oncology at Roswell Park.

Work to develop the vaccine has been enabled by donations from numerous individuals and corporations to the Roswell Park Alliance Foundation, including support from events like the Ride for Roswell, which will see thousands of cyclists riding routes throughout Western New York and into Canada on June 22.

“Roswell Park and all of Western New York have so much reason to be proud of SurVaxM and the team behind it,” says Roswell Park President and CEO Candace S. Johnson, PhD. “SurVaxM is one of the few treatment options for incurable brain cancer to reach late-stage clinical trials, and everyone who supports this work is helping to speed a highly promising cancer treatment to patients who may benefit from it.”

Last fall, the FDA awarded MimiVax and SurVaxM Fast Track Designation (FTD), a distinction that facilitates the development and review of drugs to treat serious medical conditions and fulfil unmet need. Results from the fully accrued phase 2b SURVIVE trial (NCT05163080) of SurVaxM in patients with newly diagnosed glioblastoma at 11 cancer centers nationwide are expected as early as next year, and a pilot study of the vaccine in children and adolescents with several forms of brain cancer is underway. SurVaxM is also being studied as a treatment for neuroendocrine tumors and multiple myeloma in adults.

MimiVax was formed more than a decade ago as a spinoff from Roswell Park to develop and commercialize SurVaxM.