NurExone Biologic's ExoPTEN, a therapy designed for acute spinal cord injuries, has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA). This designation offers NurExone incentives, including ten years of market exclusivity upon approval, access to grants from the European Commission and member states, and reduced-cost scientific advice to streamline the regulatory process and reduce development costs.
The EMA's decision follows the Orphan Drug Designation granted to ExoPTEN by the FDA in 2023. The FDA's designation includes tax credits for qualified clinical trials and potential seven years of market exclusivity after approval.
Preclinical Evidence
NurExone's enthusiasm stems from initial test results of ExoPTEN. Research conducted between January 2017 and May 2020 at the university level involved intranasal administration of exosomes derived from mesenchymal stem cells loaded with siRNA. Testing, which targeted complete spinal cord transection in rats, demonstrated significant functional recovery. Additional preclinical studies showed that intranasal administration of ExoPTEN led to motor improvement, sensory recovery, and faster urinary reflex restoration.
Market Potential
With approximately 50,000 new cases of acute spinal cord injuries globally each year, ExoPTEN addresses a substantial market with patients seeking to improve their quality of life. NurExone has an exclusive worldwide license from the Technion and Tel Aviv University to develop and commercialize the technology, including a patent application. The company has also developed its own intellectual property, with five families of patents.
