The US Food and Drug Administration (FDA) granted orphan drug status to SurVaxM for patients with glioblastoma.1

SurVaxM is a vaccine that targets survivin, a cell-survival protein found in most cancers.

The FDA based its approval on interim data from an ongoing phase 2 study (ClinicalTrials.gov Identifier: NCT02455557). Researchers enrolled 50 patients to receive SurVaxM in 4 doses over the first 8 weeks of treatment with temozolomide, the standard treatment for glioblastoma. Patients will continue to receive the vaccine once every 12 weeks.

Results from the IT-09 phase 1 trial showed that 7 of 8 patients who had previously failed standard therapy for recurrent malignant glioma survived for more than a year, and 3 patients maintained stable disease after more than 12 months of follow up. The average life expectancy of patients with glioblastoma receiving standard therapy alone is 7 months.2

SurVaxM was well-tolerated and patients primarily experienced grade 1 adverse events (AEs), including erythema, fatigue, myalgia, lymphopenia, and leukopenia. The only grade 3 AE observed was seizure, which was determined not to be treatment-related.

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SurVaxM is also being evaluated as a potential part of combination therapy for multiple myeloma.

Reference

  1. FDA awards orphan status to brain cancer vaccine developed at Roswell Park cancer institute [news release]. Buffalo, NY: MimiVax; August 7, 2017.  https://www.roswellpark.org/media/news/fda-awards-orphan-status-brain-cancer-vaccine-developed-roswell-park-cancer-institute. Accessed August 7, 2017
  2. Fenstermaker R, Mechtler L, Qiu J, et al. IT-09 phase I study of safety, tolerability and immunologic effects of a surviving peptide mimic vaccine (SurVaxM) in patients with recurrent malignant glioma. Neuro Oncol. 2014;16:110-8. doi: 10.1093/neuonc/nou258