NurExone Biologic Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, recognizing the potential of this regenerative therapy for acute spinal cord injury, a condition where effective treatments are limitedi. The orphan drug designation provides significant benefits to pharmaceutical companies developing drugs for rare diseases, i.e. those impacting fewer than 200,000 people in the United Statesii. These benefits include market exclusivity, financial incentives, regulatory assistance, and support with drug development.

Overall, the designation incentivizes and supports the development of certain treatments, increasing access to therapies for patients. Earning orphan-drug designation is a significant milestone for the Company. The designation covers the use of mesenchymal stem cell (MSC) derived small extracellular vesicles (EVs) loaded with short and modified interfering RNA (siRNA) targeting the phosphatase and tensin homolog (PTEN) protein for acute spinal cord injury, as implemented in the Company's ExoPTEN drug under development.