Pharma Focus Europe

FDA Grants Orphan Drug Designation to NeuExcell's AAV Gene Therapy for Malignant Glioma

Tuesday, December 19, 2023

NeuExcell Therapeutics Inc. has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting Orphan Drug Designation (ODD) to their investigational AAV gene therapy product, NXL-004. This achievement is notable as it is the first AAV gene therapy product globally designed for treating malignant glioma to receive FDA ODD designation.

Malignant gliomas, particularly glioblastoma (GBM), are the most common primary malignant brain tumors in adults. They have a high level of malignancy and associated mortality. The current standard-of-care for GBM, involving surgery, radiotherapy, and chemotherapy, results in a median overall survival of 15-18 months, with a 5-year survival rate below 10%. Recurrence of malignant gliomas is nearly inevitable, and there is currently no established standard salvage therapy.

NXL-004 represents an innovative treatment approach, utilizing the astrocyte-to-neuron ("AtN") conversion platform developed by Professor Gong Chen. Preclinical studies have shown promising efficacy and safety for NXL-004, which is set to enter first-in-human clinical trials in early 2024.

The Orphan Drug Act in the U.S. provides essential regulatory support for drugs that receive ODD, including tax credits for clinical trial expenses, exemption from new drug application fees, and a 7-year market exclusivity period post-approval. This designation is expected to accelerate the development of NXL-004, offering renewed hope for glioblastoma patients globally.

NeuExcell is dedicated to improving the health and quality of life for millions of patients globally facing neurodegenerative diseases and neurological injuries. Leveraging the transformative AtN technology, NeuExcell aims to regenerate neuronal tissues and restore neurological functions. The company's lead clinical programs, including Alzheimer's disease, ischemic stroke, and GBM, are scheduled to enter clinical trials in 2024. NeuExcell also envisions expanding its pipeline to address various indications such as Huntington's disease and Amyotrophic Lateral Sclerosis.



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