FDA Grants Orphan Drug to Anavex for Dementia

FDA has granted orphan drug designation to Anavex Life Sciences Corp. for its drug candidate Anavex 3-71 for treating frontotemporal dementia (FTD), described as a syndrome associated with progressive shrinking of the frontal and temporal anterior lobes of the brain, causing a decline in language skills and/or behavioral changes. These changes, according to the company, include inappropriate social conduct, lack of empathy, blunted emotions or agitation, neglect of personal hygiene and a decrease in energy and motivation. Anavex says that about 50,000-60,000 patients in the U.S. have FTD and there is no treatment that has been shown to slow its progression. The drug has a novel mechanism of action via sigma-1 receptor activation and M1 muscarinic allosteric modulation, which has shown to enhance neuroprotection and cognition in Alzheimer’s disease, it says.