Date: 2015-12-28
Type of information: Granting of a Fast Track status
Product name: OpRegen®
Compound: cell-based therapeutic product consisting of retinal pigment epithelial (RPE) cells
Therapeutic area: Ophtalmological diseases
Action mechanism: cell therapy. OpRegen® consists of retinal pigment epithelial cells (RPE) derived from human embryonic stem cells using a proprietary directed differentiation method. OpRegen® is formulated as a suspension of RPE cells. Preclinical studies in mice have shown that OpRegen® transplanted subretinally as a suspension of cells can rapidly organize into their natural monolayer structure and survive throughout the lifetime of the animal. OpRegen® will be an \"off-the-shelf\" allogeneic product provided to retinal surgeons in a final formulation ready for transplantation. Unlike treatments that require multiple injections into the eye, such as currently-marketed products like Lucentis and Eylea for wet-AMD, it is expected that OpRegen® will be administered in a single procedure.
Company: BioTime (USA - CA) Cell Cure Neurosciences (Israel)
Disease: age-related macular degeneration (AMD)
Latest news: * On September 28, 2015, BioTime and its subsidiary Cell Cure Neurosciences announced that the FDA has granted Fast Track designation for OpRegen®, a cell-based therapeutic product consisting of retinal pigment epithelial (RPE) cells designed to block the progression of the severe dry-form of age-related macular degeneration (AMD), a leading cause of blindness in an aging population. Under an Investigational New Drug Application (IND) for \"Retinal Pigment Epithelium (RPE) Cells derived from Allogenic Human Embryonic Stem Cells; Transplanted Subretinally\" and after receiving approval from the Israel Ministry of Health , Cell Cure is now enrolling patients at Hadassah University Medical Center in Jerusalem, Israel , in a clinical Phase I/IIa dose-escalation study evaluating the safety and efficacy of OpRegen® for geographic atrophy (GA), the severe stage of the dry form of age-related macular degeneration (dry-AMD). The first patient was treated earlier this year and Cell Cure expects to provide interim data in early 2016.
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Pediatric exclusivity UE: OTC status: Other news:
