Medical Reviewer Pharmaceuticals and Medical Devices Agency (PMDA) Tokyo, Japan
Abstract: Regenerative medicine has emerged as a transformative solution for addressing unmet needs in ophthalmology, leveraging the body's innate ability to repair and replace pathologically affected tissues. Pharmaceuticals and Medical Devices Agency (PMDA, Japan) have approved five notable regenerative medical products: Vyznova (allogeneic corneal endothelial cells for bullous keratopathy), Luxturna (gene therapy using adeno-associated virus for biallelic RPE65 mutation-associated inherited retinal dystrophy), and Nepic, Ocural, and Sakracy (autologous or oral mucosa-derived epithelial cell sheets for limbal stem cell deficiency). We analyze the latest points of review, dosage and administration, clinical data package, overview of Phase 3 clinical trials, efficacy and safety evaluation, and primary endpoints. In the development of new products, it is necessary to demonstrate the risk-benefit assessment based on the efficacy and safety of appropriate studies while capturing the features of the product and based on the basic information on the target disease. In particular, in the development of new products, it is necessary to implement the study design and safety measures based on the characteristics of the target disease and the formulation, and it is important to be able to market a useful product with the cooperation of the relevant stakeholders. The presentation will give the current status and future perspective of regenerative medical products in Ophthalmology from experiences of the PMDA.
