FDA grants orphan drug status to Anavex 2-73 to treat infantile spasms

It gives Anavex a seven year window of commercial exclusivity in the US in the area of infantile spasms, prioritized consultation by the FDA on clinical studies and exemptions on several regulatory fees.

Anavex regulatory affairs vice president Kristina Capiak said: "Infantile spasms marks the second Orphan Drug Designation for our ANAVEX 2-73 program, following the Orphan Drug Designation received for the treatment of Rett syndrome."

An infantile spasm is a particular type of seizure seen in an epilepsy syndrome of infancy and childhood known as West Syndrome.

It usually occurs in the first year of life, typically between 4-8 months.

Anavex 2-73 is an orally available drug candidate developed to potentially modify Alzheimer’s disease instead of temporarily addressing its symptoms.

It has a clean phase 1 data profile and shows reversal of memory loss and neuroprotection in various models of Alzheimer’s disease.

The company completed a phase 1 randomized, placebo-controlled single ascending dose human clinical trial of Anavex 2-73 in healthy human volunteers.

Healthy male volunteers aged 18 to 55 received single, ascending oral doses over the course of the trial, whose objectives were to define the maximum tolerated dose, evaluate pharmacokinetics (PK), clinical and lab safety.