Have you ever heard of Orphan Drug designation? In the United States, this designation is granted to drugs or biologics that intend to treat, prevent, or diagnose rare diseases – conditions affecting fewer than 200,000 Americans. As an incentive, drug developers also receive fee waivers, tax credits, and seven years of market exclusivity upon drug approval. In an article for OncLive, Chris Ryan reports that VBI-1901, an immunotherapeutic vaccine candidate for glioblastoma developed by VBI Vaccines, Inc., recently earned Orphan Drug designation. 

Evaluating a Novel Vaccine Candidate: VBI-1901

Precision Vaccinations describes VBI-1901 as: 

a novel bivalent gB/pp65 immunotherapeutic cancer vaccine candidate [in which the] enveloped virus-like particle (eVLP) technology targets two highly immunogenic cytomegalovirus (CMV) antigens. VBI-1901 is designed to coax the body to produce antibodies and white blood cells to kill tumor cells with these antigens.

While CMV can be found in many different solid tumors, an estimated 90% of glioblastomas show signs of CMV. Therefore, VBI-1901 could be incredibly effective in stimulating immune response and preventing tumor growth. 

The Orphan Drug designation comes following data from a Phase 2a clinical trial which evaluated VBI-1901 for recurrent glioblastoma. Altogether, 20 patients enrolled. These patients were then split into two cohorts. The first cohort received 10 µg of VBI-1901 alongside 200 µg granulocyte-macrophage colony-stimulating factor. This treatment led to an overall survival rate of 40% at the 18-month follow-up point. Alternately, the second cohort received 10 µg of VBI-1901 alongside ASO1B, which led to an 18% survival rate. Regardless, the data highlighted how VBI-1901 could potentially benefit those with glioblastoma without any associated toxicity issues. 

About Glioblastoma

Glioblastoma is a rare form of astrocytoma, or cancer that forms in star-shaped astrocyte cells in the brain. Doctors are not sure of what causes this cancer in every case. However, having previous radiation therapy or pre-existing genetic disorders can increase the risk. Men are more likely to develop glioblastomas than women. These tumors are extremely malignant as they are capable of making their own blood supply. Symptoms can include:

• Nausea and vomiting
• Persistent headaches
• Double or blurred vision
• Difficulty thinking or speaking
• Changes in mood, behavior, or personality
• Seizures