The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has endorsed Anavex 2-73 to receive orphan designation for the treatment of Rett syndrome, according to Anavex Life Sciences, the therapy’s developer.
The proposal will now be evaluated by the European Commission, which will take into consideration COMP’s positive opinion for its final decision, expected to be announced within a month.
If granted, the orphan medicinal product designation will provide regulatory and financial support to expedite the clinical development of Anavex 2-73 and its marketing approval in the European Union for this indication.
Anavex 2-73 has already received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Rett syndrome.
“Anavex 2-73 has the potential to provide patients and physicians with a much-needed treatment option for Rett syndrome,” Christopher U. Missling, PhD, president and chief executive officer of Anavex, said in a press release. “The COMP’s adoption of a positive opinion for Anavex 2-73 orphan drug designation is another important milestone for this program, which continues to advance rapidly.”
Anavex 2-73 is an engineered small molecule that can modulate several cellular processes relevant to neurodegenerative and neurodevelopmental diseases through the activation of the sigma-1 receptor (S1R). The investigational medicine is thought to help restore cellular balance by targeting failed protein folding, oxidative stress, mitochondrial dysfunction, and inflammation. Oxidative stress refers to cellular damage as a consequence of high levels of oxidant molecules.
Preclinical studies have shown that treatment with Anavex 2-73 can significantly improve motor and reflex response to external auditory stimulation in a mouse model of Rett syndrome. Long-term treatment over 6.5 weeks also led to improvements in balance, gait, motor learning, and muscle strength.
Two Phase 2 (NCT02756858 and 2017-004335-36) studies, in Alzheimer’s and Parkinson’s, are exploring the therapy’s potential to improve patients’ abilities to perform daily activities and prevent cognitive decline.
In animal models, Anavex 2-73 was also shown to have anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant effects, suggesting its potential to treat other neurologic disorders, such as epilepsy.