FDA Grants Orphan Drug Designation to Rett Syndrome Treatment


Anavex 2-73, which is already in clinical trials for Alzheimer’s disease, recently received Orphan Drug Designation from the FDA for a rare genetic neurodevelopmental disorder.

The FDA recently granted Orphan Drug Designation to Anavex 2-73 for treating Rett Syndrome.

Rett syndrome is a rare genetic neurodevelopmental disorder that mainly effects females and can lead to impairments in a child’s ability to speak, walk, eat, and even breathe. There is currently no cure, but symptoms can be managed through a multidisciplinary approach.

The disorder is caused by mutations in the MECP2 gene, and occurs in every 1 in 10,000 to 15,000 female live births, according to Anavex Life Sciences Corporation.

Anavex 2-73, which is currently in a phase 2a clinical trial for Alzheimer’s disease, targets sigma-1 and muscarinic receptors.

The drug showed the potential to stop and reverse Alzheimer’s disease in preclinical trials. It also showed potential to act as an anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant in animal models, according to manufacturer Anavex Life Sciences Corp.

“The Orphan Drug Designation marks the first US 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,” said Christopher U. Missling, PhD, president and chief executive officer of Anavex Life Sciences Corp. “The acknowledgement from the Agency for this orphan designation allows us to formally move forward with the planned human clinical study in this disease indication, thereby potentially expanding the reach for ANAVEX 2-73 in addition to Alzheimer’s disease.”

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