FDA awards orphan status to new brain cancer vaccine
pharmafile | August 9, 2017 | News story | Research and Development, Sales and Marketing | FDA, life sciences, orphan drug, pharma, pharmaceuticals
The FDA has announced that it has awarded orphan drug status to the vaccine SurVaxM for patients with glioblastoma, the most common form of brain cancer and a condition which affects 14,000 new patients in the US every month.
Orphan status is only awarded to innovative treatments in rare diseases, in order to facilitate innovative approaches in tackling them. The treatment works by targeting and killing survivin, a cell-survival protein found in cancer which enables the disease to resist conventional therapy methods. The vaccine was developed at Roswell Park Cancer Institute by Michael Ciesielski, Assistant Professor of Neurosurgery and Robert Fenstermaker, Chair of Neurosurgery, and both serve as executives in Roswell Park spinoff MimiVax.
“We are excited by the results to date and appreciative of this acknowledgement that SurVaxM holds promise,” explained Fenstermaker. “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 multicentre randomized study with the momentum provided by the orphan status designation.”
“There are a couple of things that distinguish our approach,” added Ciesielski. “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 vaccine is currently being evaluated for efficacy and safety in a Phase 2 trial at Roswell Park, Dana-Farber Cancer Institute, the Cleveland Clinic, Massachusetts General and Beth Israel Deaconess Medical Center. The study is assessing the therapy’s use in 50 patients during the first eight weeks of treatment with standard-of-care temozolomide, receiving the vaccine every 12 weeks.
Matt Fellows
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