VBI Vaccines Inc (NASDAQ: VBIV) has been awarded an Orphan Drug Designation by the US FDA for its bivalent GB/pp65 immunotherapeutic vaccine candidate, VBI-1901 for glioblastoma (GBM) treatment. In addition, in June 2021, the Food and Drug Administration granted the product a Fast Track Designation for recurrent GBM treatment for individuals having their first tumor recurrence.
Orphan Drug Designation to give the company market exclusivity
The Orphan Drug Designation is another milestone for the VBI-1901 program and shows the urgency of the company’s effort to create meaningful treatment alternatives for patients with GBM.
Investigational pharmaceuticals and biologics developed to prevent, diagnose, or treating uncommon medical problems or diseases that afflict less than 200,000 individuals in the United States are given the orphan drug designation by the FDA’s Office of Orphan Products Development. Pharma companies profit from the orphan drug category, which may also result in 7 years of post-approval commercial exclusivity, tax incentives for appropriate clinical studies, and waivers from some FDA expenses.
VBI presented data on VBI-1901 at ASCO
CEO of VBI Jeff Baxter “As recently presented at ASCO, we continue to see strong tumor response data and improvements in overall survival data compared to historical controls in the Phase 2a study of VBI-1901. With this orphan drug status, we look forward to working closely with the FDA and clinical investigators to build on that data, advancing the potential of this program to be a valuable part of the fight against GBM.”
Although it is an uncommon condition, GBM is the most frequent main brain cancer, with close to 14,000 new cases identified each year in the US and a short mean overall survival of 15 to18 months following the first GBM diagnosis. Stage IV tumors called glioblastomas are extremely aggressive forms of brain cancer with a significant risk of progression and a 10% five-year chance of survival. Radiation therapy, chemotherapy, and surgical resection are all considered the mainstay of treatment in the primary setting. In the recurrent situation, there is no viable standard of care; the average overall survival time for this patient cohort is about eight months.