FDA Grants Orphan Status To MabVax's Neuroblastoma Vaccine
Clinical stage oncology drug development firm MabVax Therapeutics announced that it has received the coveted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its vaccine targeting childhood cancer neuroblastoma.
The company’s neuroblastoma vaccine is indicated for patients with relapsed or recurrent high-risk neuroblastoma in remission or those with limited residual disease following the best available treatment. It is a bivalent vaccine that induces an antibody response against the two most common antigens on neuroblastoma cells. The vaccine aims to kill remaining cancer cells that can promote cancer recurrence. MabVax said it expects to start a Phase 2 trial for the vaccine in 2015.
Neuroblastoma is an extracranial solid tumor cancer in children. The rare disease is designated orphan as only 650 to 800 cases are diagnosed every year in North America. Neuroblastoma accounts for 12 percent of all cancer deaths in children younger than 15 years old. Those who survive, in particular those who have undergone multimodality therapy, suffer from severe long term outcomes, including blindness and paralysis.
J. David Hansen, CEO of MabVax, said, “There is a significant medical need to develop new therapies aimed at preventing recurrent disease in this childhood cancer. The orphan drug designation provides MabVax with substantial development incentives such as exclusivity, tax credits, PDUFA fee exemptions, and the opportunity to apply for grants to offset clinical expenses. The designation also allows the Office of Orphan Products Development and MabVax to collaborate more closely to facilitate development of the vaccine.”
A Phase 1 trial conducted at the Memorial Sloan-Kettering Cancer Center show encouraging results for the vaccine. The company has earlier received a Phase 1 Small Business Innovation Research Grant (SBIR) from the National Cancer Institute (NCI) worth $125,000 in support of the vaccine’s manufacturing and testing.
Earlier this month, the company inked another partnership agreement with Memorial Sloan Kettering Cancer Center (MSKCC) and Juno Therapeutics to leverage MabVax’s antibody discovery platform for the development of anti-cancer drugs.