- The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Anavex Life Sciences' ( NASDAQ: AVXL ) ANAVEX2-73 (blarcamesine) to treat Fragile X syndrome (FXS).
- FXS is a genetic disorder that causes developmental delays, learning disabilities and behavior problems, among other things. Autism spectrum disorder also occur more frequently in people with FXS, according to the U.S. CDC .
- The company said that currently, there is no approved treatment for Fragile X syndrome.
- "The Orphan Drug Designation highlights the potential to expand the therapeutic profile of ANAVEX®2-73 into the largest portion of autism spectrum disorder, Fragile X syndrome," said Anavex President and CEO Christopher Missling.
- The FDA grants orphan drug status to therapies which treat or prevent rare diseases that affect fewer than 200K people in the U.S. The designation provides certain incentives, including seven years of market exclusivity, if approved.
For further details see:
Anavex therapy for Fragile X syndrome gets FDA orphan drug status