Brain Cancer Vaccine SurVaxM Receives FDA Orphan Drug Status

Brain Cancer Vaccine SurVaxM Receives FDA Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to  SurVaxM, an immunotherapy treatment developed at the Roswell Park Cancer Institute for a type of brain cancer called glioblastoma.

SurVaxM, also known as DRU-2017-5947, is a novel, patented peptide immunotherapy vaccine that targets survivin, a cell-survival protein present in 95 percent of glioblastomas and other cancers. SurVaxM is engineered to recognize surviving-expressing cancer cells as foreign and stimulate the patients’ immune response to control tumor growth and recurrence.

“There are a couple of things that distinguish our approach,” Michael Ciesielski, PhD, assistant professor of neurosurgery at Roswell Park and co-inventor of SurVaxM, said in a press release. “SurVaxM is an engineered molecule capable of stimulating the immune system in several different ways to recognize and kill cancer cells. And the fact that its target, survivin, is present in many different types of cancer suggests potentially broad application against cancer.”

The orphan status application for SurVaxM was submitted by MimiVax, a Roswell Park spinoff company. The FDA’s approval notification arrived Aug. 3.

The decision was based on a Phase 2 study (NCT02455557) designed to evaluate SurVaxM in combination with standard treatment for patients with newly diagnosed glioblastoma. The trial is ongoing at Roswell Park, at the Cleveland Clinic, Dana-Farber Cancer Institute, Massachusetts General Hospital, and Beth Israel Deaconess Medical Center.

In the trial, participants received the chemotherapy Temodal (temozolomide) on days 1-5 of a 28-day cycle, followed by SurVaxM every two weeks in the first four doses, and every 12 weeks thereafter, for six courses of therapy or until disease progression or unacceptable toxicity.

“We are excited by the results to date and appreciative of this acknowledgement that SurVaxM holds promise,” said Robert Fenstermaker, MD, chair of neurosurgery at Roswell Park and co-inventor of SurVaxM. “Those of us working to help patients with glioblastoma to live longer realize that the gains from existing therapies have been quite limited. We are eager to move this work forward to a larger multicenter randomized study with the momentum provided by the orphan status designation,” he said.

For additional information about this trial, which is still recruiting participants, call (877) 275-7724), or e-mail to ASKRPCI@roswellpark.org.

SurVaxM also is being evaluated as part of a combination therapy targeting multiple myeloma in a Phase 1 clinical trial (NCT02334865), partially supported by Celgene.

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