FDA grants Orphan designation to ANAVEX 3-71 for Frontotemporal Dementia
Anavex Life Sciences, a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, has announced that the FDA granted Orphan Drug designation to ANAVEX 3-71 for the treatment of Frontotemporal dementia (FTD).
Frontotemporal dementia describes a clinical syndrome associated with progressive shrinking of the frontal and temporal anterior lobes of the brain. It causes a decline in language and/or behavioral changes, which include inappropriate social conduct, lack of empathy, blunted emotions or agitation, neglect of personal hygiene and a decrease in energy and motivation. To date, no treatment has been shown to slow its progression and the prognosis for people with FTD is poor. FTD afflicts an estimated 50,000 to 60,000 patients in the U.S. and represents an estimated 10 to 20% of all dementia cases.
“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.
FDA Orphan Drug designation is evaluated for drugs from all classes that are intended for the treatment of rare diseases, defined as diseases affecting fewer than 200,000 people in the United States. The designation provides sponsors with development and commercial incentives, including seven years of market exclusivity in the U.S., prioritized consultation by FDA on clinical studies and certain exemptions from or reductions in regulatory fees.
“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, Ph.D., president and chief executive officer of Anavex.