BioTime, Inc., a pioneer in regenerative medicine, and its subsidiary Cell Cure Neurosciences Ltd. (Cell Cure) announced that the US Food and Drug Administration (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.

The FDA grants Fast Track designation if it determines that a drug fills an unmet medical need in a serious condition. According to the FDA, a drug that receives Fast Track designation is eligible for more frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval; more frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers; eligibility for Accelerated Approval and Priority Review, if relevant criteria are met; and Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA.

Dry AMD is the most common type of macular degeneration and affects approximately 90 per cent of people with the disorder. In the dry form, there is a loss or dysfunction of the layer of retinal pigment epithelial (RPE) cells, generally in the region of the eye called the macula. These RPE cells support the light detecting photoreceptor cells that are so critical to vision. When we look at something, the photoreceptors (rods and cones) detect the light and send the information to the brain allowing us to perceive our surroundings. The age-dependent loss of the RPE cells therefore leads to degeneration of nearby photoreceptors and this can lead to severe vision loss or even blindness. Generally, the damage caused by the “dry” form is not as severe or rapid as that of the “wet” form. However, over time, it can cause profound vision loss. The more advanced stage of dry macular degeneration is called geographic atrophy. While there are therapeutics available to treat the wet form of AMD, there are currently no FDA-approved therapies for dry-AMD.