FDA Grants Orphan Drug Designation to Bivalent Vaccine for Neuroblastoma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to MabVax Therapeutics’ vaccine for the treatment of relapsed or recurrent high-risk neuroblastoma in remission or with limited residual disease after best available treatment. The bivalent vaccine is intended to elicit a targeted immune response against the two most common antigens on neuroblastoma cells, GD2 and GD3.

Results of a recent phase I trial published in Clinical Cancer Research showed encouraging outcomes with the vaccine in a small cohort of patients with relapsed neuroblastoma.¹ Antibody responses against GD2 and/or GD3 were seen in 12 of 15 patients, and disappearance of minimal residual disease was documented in 6 of 10 patients assessable for response.  ■

Reference

1. Kushner BH, Cheung IY, Modak S, et al: Phase I trial of a bivalent gangliosides vaccine in combination with β-glucan for high-risk neuroblastoma in second or later remission. Clin Cancer Res 20:1375-1382, 2014.