FDA Grants Orphan Drug Designation to AAV Gene Therapy Product for Malignant Glioma

NXL-004 (NeuExcell Therapeutics, Philadelphia, PA) has been granted Orphan Drug Designation (ODD) by the Food and Drug Administration (FDA). NXL-004 is the first investigational adeno-associated virus vector (AAV) gene therapy product developed as a potential treatment for malignant glioma to receive ODD from the FDA.

NXL-004 is based on the in vivo astrocyte-to-neuron (AtN) conversion platform developed by Gong Chen, PhD, Cofounder and Chief Scientific Officer of NeuExcell. According to company statements, this AtN conversion platform has the potential to regenerate the damaged neuronal tissue present in neurodegenerative conditions, traumatic brain injury (TBI), spinal cord injury, and glioma. NeuExcell Therapeutics notes that NXL-004 has shown positive efficacy results in preclinical trials, along with a favorable safety profile, and the company plans to initiate first-in-human clinical trials in early 2024.

Malignant gliomas are cancerous brain tumors, approximately 60% of which are caused by glioblastoma (GBM), the most common type of cancer originating in the brain. A diagnosis with GBM is characterized by high malignancy and mortality and is associated with a 15-to-18-month overall survival and a 5-year survival rate under 10%.

The FDA grants orphan drug designation to agents that prevent, diagnose, or treat rare diseases and conditions with the aim of supporting their clinical development and evaluation.