Blarcamesine, also known as Anavex 2-73, previously secured orphan drug designation from the FDA, a status intended to incentivize the development of treatments for rare diseases. The company now holds active investigational new drug applications across three primary therapeutic areas: Alzheimer’s disease, Rett syndrome, and Fragile X syndrome.

Interim CEO Terrie Kellmeyer described the clearance as a significant milestone, noting that the company intends to maintain its current momentum in clinical development. The study marks a shift in the biotech firm’s efforts to address the neurological manifestations associated with the syndrome, as leadership continues its collaborative approach with federal regulators to advance their broader pipeline.