Orphan Drugs are defined by the FDA as drugs and biologics which are intended for the treatment, prevention, or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.
Verax's Primary Orphan Drug Focus: Glioblastoma
Of all types of gliomas, glioblastoma multiforme (GBM), a grade 4 brain tumor, is both the most common and most aggressive malignant, nonhereditary tumor of the brain. The prognosis for patients with GBM is grim, with a survival rate of 12-18 months, and only 25% of patients surviving over 1 year and 5% surviving over 5 years despite treatment. Standard treatment for GBM is surgical resection, radiation therapy, and chemotherapy, yet tumor recurrence occurs in most patients and other treatment options are mostly palliative in nature.
In the United States, approximately 200,000 people currently have this condition, which qualifies any treatment for GBM to be in the Orphan Drug designation. Since 1984, there have been 72 Orphan Drug designations for GBM, each with a focus on eradicating cancer. However, none of the products designated have ever received approval for commercial use. We believe a non-invasive and non-toxic organic ingredient approach to reducing the tumor size has a much higher probability of FDA approval.
Clinicians and patients see enormous potential in novel approaches and improvement in the delivery of antiproliferative therapies. A recently published study recognizes the use of inhaled CBD as a promising GBM treatment. A quote from the article cited provides some interesting insights, “The latest significant advance in treating glioblastoma came more than 15 years ago, with the addition of the chemotherapy drug temozolamide to radiation, which extended survival about one- and one-half months and was celebrated by patients and caregivers alike.” Preliminary data shows CBD's role as an immune regulator, with the ability among other mechanisms to suppress IL-8, which is secreted by glioblastoma cells. This market is clearly ready for any innovation.
Verax and our scientific partners have been working on an inhaled Nitrous Oxide CBD (non-THC) product for the treatment of GBM. Our vision is to transform Glioblastoma into a manageable condition and give patients more quality time post-diagnosis.
Other possible orphan designations in our portfolio include:
Hyper-Immunoglobulin M Syndromes (HIGM),
PLCG2-associated Antibody Deficiency and Immune Dysregulation (PLAID),
Wiskott-Aldrich Syndrome (WAS)
Hyper-Immunoglobulin E Syndrome (HIES)