Orphan Drug Designation Granted to Frontotemporal Dementia Treatment

The FDA recently granted Orphan Drug Designation (ODD) to Anavex 3-71 for the treatment of Frontotemporal dementia (FTD), a disease that affects 50,000 to 60,000 patients in the United States.

“We believe that Orphan Drug Designation for ANAVEX 3-71 for the treatment of Frontotemporal dementia is a significant achievement,” said Kristina M. Capiak, Vice President of Regulatory Affairs at Anavex.

Dementia is associated with the progressive shrinking of the frontal and temporal anterior lobes of the brain. Individuals will experience a decline in language as well as behavioral changes.

These behavioral issues include inappropriate social conduct, lack of empathy, blunted emotions and agitation, decreased personal hygiene and diminished energy.

At this time there are no treatments that have proven to be effective in slowing down the progression of FTD.

ANAVEX 3-71 targets the sigma-1 and M1 muscarinic receptors and demonstrates neuroprotection and improved cognition in Alzheimer’s disease models. The CNS-penetrable monotherapy is able to treat cognitive impairments with potential disease modifications.

The drug has been found effective in very low doses for the significant Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, such as cognitive deficits, amyloid and tau pathologies. It has also shown positive effects on neuroinflammation and mitochondrial dysfunctions.

“This regulatory milestone allows us to continue to advance in the rare disease space while expanding our portfolio within neurodegenerative diseases,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.

The Clinical Focus condition center at NeurologyLive, Specialty Pharmacy Times' new sister site, provides further coverage from the Alzheimer’s disease space, as well as updates pertaining to dementia and the most recently released data from the field’s most prominent conferences.