FDA Grants Orphan Drug Designation to Exosome-Based Therapy for Glioblastoma Multiforme

The FDA has granted orphan drug designation to an exosome-based therapy as a potential therapeutic option for patients with glioblastoma multiforme (GBM), according to an announcement from Exousia Pro.1

“GBM is the most common and highly malignant central nervous system [CNS] tumor that currently lacks adequate treatment," Marvin S. Hausman, MD, chairman of the Scientific Advisory Board at Exousia Pro, stated in a 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.”

The exosome-based therapy is not currently being evaluated in patients with GBM in any clinical trials, and the orphan drug designation was supported by preclinical data for the treatment.

"The Company is very excited to have been granted this designation," stated Matthew Dwyer, president of Exousia Pro, stated in a news release. "It is hard for us to quantify the value of orphan drug designation…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.”

How was exosome-based therapy evaluated preclinically in GBM?

The preclinical evaluation of the exosome-based therapy utilized 32 immunodeficient mice, whose brains were implanted with glioblastoma tumor cells derived from human patients.2 According to Exousia Pro, the majority of preclinical studies in glioblastoma utilize genetically engineered or chemically induced mouse models, which can restrict the translation of data to human disease. In this preclinical study, Exousia Pro implanted surgically-removed human glioblastoma tumor cells into the immunodeficient mice in order to make a clinically relevant evaluation of therapeutic efficacy of the exosome-based therapy.

Researchers allowed for the tumors to establish in the mice for 14 days before they were randomly assigned to receive conventional temozolomide alone; exosome treatment alone; a combination of temozolomide and exosome therapy; or no treatment (control).

The mice underwent 7 days of treatment before being evaluated for tumor size and body weight changes to assess the efficacy of the therapies and the overall health of the mice. The company stated that data on tumor size reduction would be critical for supporting the orphan drug designation application, and immunohistochemical analyses of brain slices would provide further evidence of the effect of exosome-based therapy.

"This preclinical trial could alter the trajectory of exosomal-based therapies, which [have] seen relatively low adoption due to production and manufacturing inefficiencies," Mike Sheikh, chief executive officer of Exousia Pro, stated in an April 2025 news release. "We can target and load the exosome in a singular process whereby the cell does all the work for us. Our in vitro studies demonstrated that our exosomal therapy, combined with minimal chemotherapy, significantly enhances the elimination of cancer stem cells—the primary barrier to effective GBM therapy. If our preclinical results validate or exceed these findings, Exousia Pro could achieve global recognition and transform the standard of care for [patients with GBM].”

References

1. Exousia Pro is excited to announce that it has received orphan drug designation from the FDA. News release. Exousia Pro. October 28, 2025. Accessed October 29, 2025. https://www.otcmarkets.com/stock/MAJI/news/Exousia-Pro-is-Excited-to-Announce-that-it-has-Received-Orphan-Drug-Designation-from-the-FDA?id=497771
2. Exousia Pro finishes preclinical trial for glioblastoma ahead of schedule - bolstering orphan drug application. News release. Exousia Pro. April 29, 2025. Accessed October 29, 2025. https://www.otcmarkets.com/stock/MAJI/news/Exousia-Pro-Finishes-Preclinical-Trial-for-Glioblastoma-Ahead-of-Schedule---Bolstering-Orphan-Drug-Application?id=475208