Pharma Focus Asia

NurExone Biologic Secures FDA Orphan-Drug Designation to Expedite Development of ExoPTEN Therapy for Treating Acute Spinal Cord Injuries

Tuesday, October 31, 2023

NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V), a leading biotechnology company, is delighted to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for its innovative therapy, ExoPTEN. This designation recognizes the therapeutic potential of ExoPTEN for acute spinal cord injury, a condition with limited treatment options.

Orphan drug designation provides valuable benefits to pharmaceutical companies working on treatments for rare diseases, which are defined as those affecting fewer than 200,000 people in the United States. These benefits encompass market exclusivity, financial incentives, regulatory support, and assistance in the drug development process. Overall, this designation encourages and supports the development of specific therapies, ultimately increasing access to treatments for patients.

Securing orphan-drug designation is a significant achievement for NurExone. This designation pertains to the use of small extracellular vesicles (EVs) derived from mesenchymal stem cells (MSCs) loaded with short and modified interfering RNA (siRNA) targeting the phosphatase and tensin homolog (PTEN) protein for the treatment of acute spinal cord injury, as incorporated in NurExone's ExoPTEN drug, which is currently in development.

"Obtaining orphan-drug designation is expected to simplify our path to market, shorten the regulatory process, resulting in substantial cost savings for the company, and grant valuable market exclusivity. We are grateful for the official recognition of the potential impact of our therapy on the lives of patients coping with acute spinal cord injuries," stated Dr. Shaltiel, CEO of NurExone Biologic, Ltd.



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