Anavex gets orphan drug status in Rett syndrome

Anavex Life Sciences

The FDA has granted orphan drug designation to Anavex Life Sciences’ (NASDAQ:AVXL) ANAVEX 2-73 for the treatment of Rett syndrome.

In a statement, Christopher Missling, president and CEO, said orphan drug designation marks the first U.S. movement for ANAVEX 2-73.

Rett syndrome is a devastating disease occurring in early childhood and almost exclusively in girls.  As there is currently no cure and limited treatment, Rett syndrome represents a clear unmet clinical need.

Orphan drug designation also allows Anavex to move forward with a planned human clinical study in Rett syndrome, “potentially expanding the reach for ANAVEX 2-73 in addition to Alzheimer’s disease,” Mr. Missling added.

Sign up for the BioTuesdays weekly newsletter

Get the latest news on the healthcare industry’s corporate and clinical developments, executive moves, and market updates. Every Tuesday, in your inbox.