NurExone Biologic Inc. has secured Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for its ExoPTEN therapy, a significant step toward providing a new treatment option for acute spinal cord injury patients in Europe. The designation offers substantial benefits, including a decade of market exclusivity upon approval, access to grants, and reduced expenses for scientific consultation.
Addressing a Critical Unmet Need
The EMA estimates approximately 20,000 new cases of acute spinal cord injury occur annually within the European Union. These injuries often lead to lifelong care requirements, with limited effective therapeutic options currently available. ExoPTEN aims to address this gap by promoting nerve regeneration and functional recovery in patients with spinal cord injuries.
ExoPTEN Mechanism of Action
ExoPTEN utilizes mesenchymal stem cell-derived extracellular vesicles loaded with siRNA targeting the PTEN protein, a key regulator of nerve regeneration. By inhibiting PTEN, ExoPTEN seeks to stimulate nerve regeneration and improve functional outcomes following spinal cord injury. Preclinical studies have demonstrated promising results, with motor function recovery observed in 75% of laboratory rats when administered intranasally.
Regulatory and Financial Incentives
The Orphan Medicinal Product Designation from the EMA not only validates the potential of ExoPTEN but also provides crucial regulatory support as NurExone prepares to advance into clinical trials. The company may also benefit from free or reduced-cost scientific advice and assistance with clinical trial design, streamlining the regulatory process and reducing development costs. Furthermore, several European Union countries offer tax credits and other financial incentives to support orphan drug development.
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 their ability to accelerate the global development of ExoPTEN and address the urgent unmet needs of patients worldwide.
Next Steps
NurExone plans to work closely with the EMA and other agencies to expedite the development of ExoPTEN and bring this innovative treatment to SCI patients across Europe. The company anticipates initiating clinical trials to further evaluate the safety and efficacy of ExoPTEN in humans.
