NurExone Biologic Inc. has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for its ExoPTEN therapy, a significant advancement towards its availability for acute spinal cord injury (SCI) treatment in Europe. This designation not only supports the development of ExoPTEN but also paves the way for faster market entry in Europe, where there is a high demand for effective SCI therapies.
ExoPTEN: A Novel Approach to Spinal Cord Injury Treatment
ExoPTEN is designed to promote nerve regeneration and functional recovery following spinal cord injury. The therapy utilizes mesenchymal stem cell-derived extracellular vesicles loaded with siRNA targeting PTEN, a key protein that inhibits nerve regeneration. By targeting PTEN, ExoPTEN aims to enhance the body's natural ability to repair damaged spinal cord tissue.
EMA Orphan Drug Designation: Benefits and Incentives
The EMA’s Orphan Medicinal Product Designation provides several valuable incentives to NurExone, including:
- Market Exclusivity: 10 years of market exclusivity upon approval.
- Financial Incentives: Access to grants and incentives from the European Commission and Member States.
- Regulatory Support: Free or reduced-cost scientific advice and assistance with clinical trial design.
- Tax Benefits: Potential tax credits and other financial incentives in some European Union countries.
Leadership Perspective
"We are honored by the EMA’s recognition of ExoPTEN through the Orphan Medicinal Product Designation, which significantly advances our ability to enter the European market and offers hope to those impacted by acute spinal cord injuries," said Dr. Lior Shaltiel, Chief Executive Officer of NurExone. He added that this designation, along with the FDA's Orphan Drug Designation, reinforces the company's ability to accelerate the global development of ExoPTEN and address the urgent unmet needs of patients worldwide.
Addressing Unmet Needs in SCI Treatment
According to the EMA, approximately 20,000 new cases of acute spinal cord injury occur in the European Union each year. These patients often require lifelong care, and effective therapeutic options are limited. ExoPTEN's innovative approach to promoting spinal cord recovery directly addresses this gap, with the potential to meet a critical need in the European healthcare system.
Regulatory Pathway
Dr. Ina Sarel, NurExone’s Head of CMC Quality and Regulation, noted that the EMA’s designation acknowledges ExoPTEN’s potential and paves the way for essential regulatory support as the company prepares to advance into clinical trials. NurExone intends to work closely with the EMA and other agencies to accelerate ExoPTEN’s development and bring this innovative treatment to SCI patients across Europe.
