Vaccine for Glioblastoma Awarded Orphan Drug Status by FDA

MimiVax announced that its immunotherapy vaccine SurVaxM, which is being tested in clinical trials for glioblastoma, has been designated an orphan drug by the FDA.

According to a press release from the Roswell Park Cancer Institute, its spinoff company MimiVax LLC was notified by the FDA on August 3 that its orphan status application for SurVaxM had been approved. The vaccine works by triggering the immune system to attack survivin, a protein present in most cancers that lets them withstand traditional treatments. Its creators hope that its multifaceted mechanisms of action will allow it to halt tumor growth and prevent recurrence.

“SurVaxM is an engineered molecule capable of stimulating the immune system in several different ways to recognize and kill cancer cells,” said Michael Ciesielski, PhD, assistant professor of neurosurgery at Roswell Park and chief scientific officer of MimiVax, in the press statement. “And the fact that its target, survivin, is present in many different types of cancer suggests potentially broad application against cancer.”

The vaccine, also called DRU-2017-5947, is currently being tested in conjunction with standard of care therapy in a phase 2, multicenter clinical trial involving patients with newly diagnosed glioblastoma. The press release indicates that the results of interim analyses have been encouraging, and the company intends to pursue further clinical trials of SurVaxM and eventual FDA approval. An ongoing phase 1 clinical trial is also testing the vaccine in patients with multiple myeloma.

“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 chief medical officer at MimiVax. “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.”

Although glioblastoma is the most common form of brain cancer seen in adults, it is still considered a rare disease, with around 14,000 new cases diagnosed annually. It generally carries a poor prognosis and has a median survival time of 15 to 18 months, according to researchers from the National Cancer Institute (NCI) who recently held a question-and-answer discussion about the cancer.

Glioblastoma has been identified as a likely target for immunotherapy innovations because it does not respond well to traditional cancer treatments. For instance, the NCI researchers explained, these tumors are heterogeneous and grow quickly, and the blood-brain barrier prevents many drugs from even reaching them.

As with any drug designated an orphan drug by the FDA, the lack of current effective treatment options means that SurVaxM could fill an important clinical gap, if approved. The Orphan Drug Act encourages innovative drug development for rare diseases through incentives like tax credits for clinical trials and waiving the prescription drug user fee that is usually collected with a drug’s marketing application.