According to a recent press release by Anavex, the United States Food and Drug Administration has approved an investigational new drug application for a study of the drug Anavex®2-73 in Patients with Rett syndrome. A Phase 2 study will now go ahead as planned.
About Rett Syndrome
Rett syndrome is a genetic disorder that causes severe mental and physical disability. It usually occurs in girls, although in rare cases males can also be affected, and, in a large majority of cases, there is no family history of the disorder. Children with Rett syndrome often show normal growth and development for around six months or longer, and then may begin to show symptoms such as slower development, changed hand movements, slowed brain growth, walking issues, seizures, and intellectual disability.
Anavex 2-73
Anavex Life Sciences is currently researching an investigational drug called Anavex®2-73 as a potential treatment for Rett syndrome. The drug, which activates the Sigma-1 receptor protein, has been researched in studies using mouse models that mimicked the symptoms of Rett syndrome. The studies showed that the drug was associated with improvements in several of the mice’s symptoms. In addition to Rett syndrome, Anavex 2-73 is also being explored as a potential treatment for Parkinson’s disease dementia and Alzheimer’s disease.
Research Updates
The FDA recently approved an investigational new drug application (IND) for Anavex 2-73 as a treatment for Rett syndrome. An IND is an exemption that allows unapproved drugs to be transported across state lines, which is often necessary in clinical trials.
Anavex is now able to go ahead with their planned Phase 2 study of Anavex 2-73, which will be a double-blind and randomised trial that compares the effects of the drug to those of a placebo in patients with Rett syndrome. According to Anavex, fifteen patients will take part in a seven-week treatment period, with an optional open-label extension study for those who want to continue treatment with the drug.
