The US FDA Grants Orphan Drug Designation to Anavex 3-71 for FTD

The US FDA has granted orphan drug designation to Anavex 3-71 to treat frontotemporal dementia. The small molecule drug, developed by Anavex Life Sciences, is an activator of sigma-1 receptors and modulator of the M1 muscarinic receptors.  In 3xTg transgenic mouse models of Alzheimer’s disease, Anavex 3-71 improved cognition, reduced amyloid and tau pathology, and reduced neuroinflammation. Anavex 2-73, the company’s lead compound, is also a sigma-1 receptor agonist and is being tested in Phase II trials in AD.

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