On December 7, 2023, Beijing time, NeuExcell Therapeutics announced that its proprietary AAV gene therapy product, NXL-004, designed for treating malignant glioma, has been awarded Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This milestone was achieved following NeuExcell Therapeutics’ application in September 2023 for NXL-004 as a treatment for Glioblastoma Multiforme (GBM). The designation received not only covers GBM but also extends to broader indications within malignant glioma, surpassing the initial scope of the application. NXL-004 marks a global first, being the inaugural malignant glioma AAV gene therapy product to secure FDA orphan drug status. PackGene Biotech, a collaborator with NeuExcell Therapeutics, extends heartfelt congratulations on this pivotal achievement.

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Gliomas, tumors that arise from the brain’s glial cells, are the most prevalent type of primary intracranial tumors. According to the 2021 WHO classification of central nervous system tumors, gliomas are graded from 1 to 4, with grades 1 and 2 categorized as low-grade gliomas and grades 3 and 4 as high-grade, or malignant gliomas. The majority of these malignant gliomas are classified as Glioblastoma Multiforme (GBM), known for its aggressive nature and high mortality rate. Despite significant advancements in the understanding and treatment of GBM, the outcomes remain limited. The current standard of care for malignant gliomas includes a combination of surgery, radiation, and chemotherapy. Clinical trials indicate that the median survival rate for GBM patients is approximately 15-18 months, with a 5-year survival rate below 10%. Relapse in GBM patients is almost inevitable, reducing the median survival period to just 24-44 weeks post-recurrence.

NXL-004 is a groundbreaking gene medication for glioma treatment, developed from the innovative in situ cell transdifferentiation technology platform pioneered by Professor Gong Chen’s team. Preclinical research has demonstrated the drug’s promising efficacy and safety. Having completed Good Manufacturing Practice (GMP) production and toxicological assessment, NXL-004 is poised to enter the clinical trial phase. Under the U.S. Orphan Drug Act, drugs granted orphan status benefit from various policy supports, including tax credits for clinical trials, exemption from new drug application fees, and an exclusive marketing period of seven years post-approval. The orphan drug status of NXL-004 is pivotal for the advancement of its pipeline, representing a beacon of hope for malignant glioma treatment.

Integral to the development of NXL-004 was the collaboration with PackGene Biotech. PackGene provided the needed GMP materials, encompassing both plasmid and AAV vectors, critical for the development of NXL-004. Their contribution extended to the analytical development of these components, ensuring compliance with the rigorous therapeutic standards. This collaboration was crucial in advancing NXL-004 towards its successful FDA Orphan Drug Designation.

 

About NeuExcell Therapeutics

NeuExcell Therapeutics, a global leader in innovative biotechnology, is dedicated to treating neurological injuries and degenerative diseases through its pioneering in situ neural regeneration technology. With subsidiaries in both China and the United States, the company was founded by Professor Gong Chen, a globally renowned expert in regenerative medicine. He is recognized for his pioneering work in the direct conversion of endogenous astrocytes into functional neurons in adult mammalian brains. NeuExcell’s executive team comprises industry veterans with extensive experience at world-renowned pharmaceutical companies, including Novartis, Pfizer, AstraZeneca, Sanofi, and Spark Therapeutics. The company’s diverse product pipeline addresses major neurological conditions, such as stroke, glioma, Alzheimer’s disease, Huntington’s disease, and amyotrophic lateral sclerosis.

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