FDA Grants Exousia Pro Orphan Drug Designation in Glioma

Exousia Pro, Inc., a clinical-stage biotech company that specializes in the use of exomes to treat cancer and other illnesses, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with malignant glioma, also known as glioblastoma multiforme or GBM.

The development was announced in a news release issued by Exousia Pro, Inc., noting that in 2024 the company’s subsidiary, Exousia AI, and Dr. Marvin S. Hausman filed for orphan drug designation for an exome-based treatment for GBM.

"GBM is the most common and highly malignant central nervous system (CNS) tumor that currently lacks adequate treatment," stated Hausman, chairman of the company’s Scientific Advisory Board, in a statement that was included in the news release. "Our breakthrough exosomal technology has the ability to deliver a wide range of therapeutics, including genetic material, into cells afflicted with cancer, such as GBM. The therapeutic technology presented in this orphan drug application is a method for using exosomes loaded with desired nucleic acids, in the effective treatment of GBM when combined with currently available standard anticancer therapy."

An exome, as defined in ScienceDirect, is the region of the genome that encodes proteins, comprising approximately 1% to 2% of the genome and containing approximately 85% of disease-causing variants.

The FDA, as the agency explains on its website, has authority to grant orphan drug designation to a drug or biological product that is intended to prevent, diagnose or treat a rare disease or condition. This designation qualifies sponsors for a number of incentives such as tax incentives for qualified clinical trials, exemption from user fees and a potential seven years of market exclusivity following a drug’s FDA approval, as the agency explains.

"The Company is very excited to have been granted this designation," said Matthew Dwyer, President of Exousia Pro, in a statement included in the news release. "It is hard for us to quantify the value of [orphan drug designation] as it is likely in the tens of millions of dollars. We will soon begin working with investment bankers to monetize the [orphan drug designation] as we advance our therapy. GBM is a terrible disease and pretty much a death sentence for most. We believe our combination therapy has the potential to be disease-modifying and could make a huge difference for this patient population."

More About Glioblastoma

Glial cells, as defined by the National Cancer Institute on its website, are any of the cells that hold nerve cells in place and help them work in the way that they should.

Glioma is not a specific type of brain tumor; it is a general term used to describe tumors that start in the glial cells such as astrocytomas, which include glioblastomas, according to the American Cancer Society, which noted on its website that approximately three out of 10 brain tumors are gliomas. High-grade astrocytomas tend to grow quickly and spread to surrounding normal brain tissues. GBMs, also known as grade 4 astrocytomas, are the fastest growing and make up more than half of all gliomas, and are the most common malignant brain tumors in adults.

Glioblastomas, according to the American Brain Tumor Association, make up approximately 14% of all primary brain tumors, and on average more than 12,000 cases of glioblastoma are diagnosed each year in the United States.

References

1. “Exousia Pro is Excited to Announce that it has Received Orphan Drug Designation from the FDA,” news release; https://www.accessnewswire.com/newsroom/en/healthcare-and-pharmaceutical/exousia-pro-is-excited-to-announce-that-it-has-received-orphan-drug-d-1092559
2. “Exome,” ScienceDirect; https://www.sciencedirect.com/topics/medicine-and-dentistry/exome
3. “Designating an Orphan Product: Drugs and Biological Products,” FDA; https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
4. “Glial cell,” National Cancer Institute; https://www.cancer.gov/publications/dictionaries/cancer-terms/def/glial-cell
5. “Types of Brain and Spinal Cord Tumors in Adults,” American Cancer Society; https://www.cancer.org/cancer/types/brain-spinal-cord-tumors-adults/about/types-of-brain-tumors.html
6. “Glioblastoma (GBM),” American Brain Tumor Association; https://www.abta.org/tumor_types/glioblastoma-gbm/

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