News
January 24, 2024 - VGN-R09b, independently developed and manufactured by Shanghai Vitalgen BioPharma Co., Ltd. and its wholly-owned subsidiary Shanghai Taichang Biotechnology Co. Ltd. (hereinafter referred to as the “Company”), has obtained the acceptance notice issued by the NMPA, with the indications to primary Parkinson's disease (PD) and aromatic L-amino acid decarboxylase deficiency (AADCD), and it is the first gene therapy variety to submit the IND application and be accepted in China.
Administrative Licensing Documents by National Medical Products Administration
Acceptance Notice
The following application made by your company:
Application: Clinical Study for Registration of Domestically Produced Drugs
Product Name: VGN-R09b
The first clinical study of VGN-R09b, Early Trial to Evaluate Tolerability, Safety, and Efficacy of VGN-R09b by striatal injection in patients with AADC deficiency, has been completed enrollment. The children's physical motor functions and intelligence improved significantly following treatment, and the children who received treatment earlier had achieved the milestone of raising their heads without support from others, and the further movement milestone of sitting alone could be expected. The existing clinical data make clinical investigators greatly confident in VGN-R09b, which is a gene therapy drug worth looking forward to.
Dr. Zhao Xiaoping, CEO of Vitalgen BioPharma, said: “Thank you to every patient and their families, investigators and participants, you together with us witnessed one after another of the children's milestones: the children who were completely paralyzed before is now able to look up, touch their mother's hair, grab toys with parents, and push away things they don't like. Some major movements common in ordinary families can make the parents excited for a long time, which also makes us deeply appreciate the sense of mission and pride in the drug development of treatment of rare diseases in children, and the practical sense of happiness and gain brought by the development of biological drugs for the ordinary people. although born with rare diseases, never give up, for every patient with rare disease, as well as us.”
The acceptance of the IND application of VGN-R09b for the treatment of Parkinson's disease marks that the Vitalgen BioPharma gene therapy products have been expanded from rare diseases to common diseases, and is an important milestone in the company's commitment to gene therapy drug development. “VGN-R09b has been rapidly validated for the efficacy based on the clinical study of rare diseases, and therefore we are more confident in the application prospects of VGN-R09b in common diseases, frequently-occurring diseases such as Parkinson's disease. Therefore, we submitted the IND applications for two indications to the NMPA at the same time. We hope that VGN-R09b will benefit more patients with its good safety and superior efficacy” said Dr. Zhao.
About AADC Deficiency
Aromatic L-amino acid decarboxylase (AADC) deficiency is caused by a pathogenic mutation in the DDC gene encoding AADC, which is responsible for the decarboxylase of L-DOPA (levodopa) and 5-HTP (5-hydroxytryptophan) to form the neurotransmitters dopamine and 5-hydroxytryptamine, respectively. AADC deficiency impedes the synthesis of critical neurotransmitters that control the sympathetic nervous system, emotion, cognition and motor coordination, presenting clinical symptoms such as motor dysfunction, mental retardation and diminished alertness and sleep disturbances, with a risk of premature death in the first decade of life. There are no formal epidemiological studies of the disease in China except a newborn screening program for AADC deficiency in Taiwan, which showed a birth incidence of AADC deficiency of approximately 1/32,000.
About Parkinson's Disease
Parkinson's disease (PD) is the second most common neurodegenerative disease, and the prevalence of Parkinson's disease is increasing faster than many other neurodegenerative diseases. Parkinson's disease is estimated to affect about 1% of the population over the age of 65 and 4% of those over the age of 85. The prevalence of people over 65 years old in China was 1.7%, which was similar to that of European and American countries. In the future, the number of patients with Parkinson's disease in China will rise from 1.99 million in 2005 to 5 million in 2030, accounting for almost half of the number of Parkinson's patients around world.
About VGN-R09b
VGN-R09b is a gene therapy product independently developed and manufactured by Vitalgen BioPharma, which uses recombinant AAV as a gene therapy vector to recover dopamine production based on the delivery of functional AADC, thus playing the corresponding potential therapeutic effects. By local striatal injection, the drug can be applied directly to the site of disease, reducing the dose administered systemically and correspondingly leading to a decrease in adverse effects such as immune reactions. Theoretically, gene therapy requires only one injection to have long-term therapeutic effects.
