FDA Orphan Drug Designation Granted to Exosome-Based Treatment for GBM

An exosome-based treatment of glioblastoma multiforme (GBM) has received orphan drug designation from the FDA. ¹ Exousia AI, a subsidiary of Exousia Pro, filed for orphan drug designation of the exosome-based GBM treatment last year.

“GBM is the most common and highly malignant central nervous system (CNS) tumor that currently lacks adequate treatment,” said Marvin S. Hausman, MD, chairman of Exousia Pro’s Scientific Advisory Board, in a press release.¹ “Our breakthrough exosomal technology has the ability to deliver a wide range of therapeutics, including genetic material, into cells affiliated 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.”

In order to receive an orphan drug designation, a company must submit a request to the FDA with a scientific rationale demonstrating a medically plausible basis for expecting the drug to be effective in treating the rare disease. Preclinical or clinical data usually support this rationale. Additionally, the status is granted to encourage development of treatments for rare diseases, which affect fewer than 200,000 people in the U.S.

In cancer diagnosis and treatment, exosomes could be used to carry tumor-associated antigens or RNA-based treatments that could target and destroy cancer cells. Exousia Pro hopes that plant exosome-based diagnostics could be developed as noninvasive tests for detecting cancer, monitoring treatment response, targeting specific cancer cells, and improving the precision of cancer therapies while reducing damage to healthy tissue.²

Exosome-based treatment can also help combat the presence of the blood-brain barrier and blood-brain tumor barrier in GBM. As a non-invasive drug delivery method, exosomes are natural drug delivery vehicles with high biological barrier penetrability.³

Another goal of exosome-based therapy is the possibility of targeted drug delivery, which could deliver therapeutic drugs, gene therapies, or vaccines directly to specific cells or tissues.²

Matthew Dwyer, president of Exousia Pro, stated in the press release that it is “hard to quantify the value of [orphan drug designation] as it is likely in the 10’s of millions of dollars.”¹ Dwyer noted that the company will begin working with investment bankers to move forward with the opportunities provided by the designation.

REFERENCES:

1.Exousia Pro is Excited to Announce that is has Received Orphan Drug Designation from the FDA. Press release. ACCESS Newswire. October 28, 2025. Accessed October 29, 2025. https://tinyurl.com/598emvu3

2.Understanding Exosomes. Exousia Pro. Accessed October 29, 2025. https://tinyurl.com/56454khz

3. Khatami SH, Karami N, Taheri-Anganeh M, et al. Exosomes: Promising Delivery Tools for Overcoming Blood-Brain Barrier and Glioblastoma Therapy. Mol Neurobiol. 2023;60(8):4659-4678. doi:10.1007/s12035-023-03365-0