FDA Grants Orphan Drug Status to SurVaxM for Glioblastoma
The US Food and Drug Administration granted orphan drug status to SurVaxM for patients with glioblastoma.
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.
- 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
- 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