Anavex Life Sciences Corp (AVXL)

[Talon38]

Gator, Not sure the RMAT is an unlikely designation for 2-73 given its possible and likely application as a drug for a very broad spectrum of CNS diseases. With clinical results for Alzheimer's and strong preclinical results for Parkinson's, M/S, Rett Syndrome, Angelman and possibly insomnia, ANAVEX 2-73 looks to be just the type of drug that the FDA would want as a poster child. Its success would demonstrate how the 21st Century Cures Act and this Administration is going to "Make America Great Again" in the pharmaceutical and health areas. It also meets the FDA priority of championing drugs that would have a significant impact on reducing drug and treatment costs for Medicare and Medicaid.

Here is the RMAT excerpt from Commissioner Gottlieb' s 7 July address: "How FDA Plans to Help Consumers Capitalize on Advances in Science". The link below provides Dr Gottlieb's entire comments and is essential reading in order to see where the FDA is going.


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"Among some of the other noteworthy actions that we’re pursuing under Cures:

Our Center for Biologics Evaluation and Research (CBER) is implementing the Regenerative Medicine Advanced Therapy, or RMAT designation. This new process provides another pathway to access FDA’s existing expedited programs, and is available for certain cell therapies, therapeutic tissue engineering products, and certain combination products. The goal of these efforts is to help foster the development and approval of these novel products. We’ve already received almost two dozen requests for RMAT designation and granted four such designations to date. To continue to advance these opportunities, we’ll be announcing this September a comprehensive framework for the development and proper FDA oversight of regenerative medicine. This new policy effort will comprise a series of new guidance documents covering many aspects of the regulation of regenerative medicine products. It will be announced as part of our Innovation Initiative. It will delineate our policies for appropriate and efficient regulatory oversight of regenerative medicine products, in order to demonstrate their safety and effectiveness. It will also create an accessible framework that will enable providers to more easily collaborate on proving these principles for regenerative products that are advanced within local medical institutions. We want to help facilitate these scientific advances, which hold out tremendous potential for treating and even curing diseases. To achieve these goals, we need to make sure that we have a modern regulatory framework in place that can allow innovators to meet the statutory requirements for demonstrating safety and effectiveness. "


https://blogs.fda.gov/fdavoice/index.php/2017/07/how-fda-plans-to-help-consumers-capitalize-on-advances-in-science/