Newron sees its Investigational New Drug application approved by the FDA. It can now begin clinical trials for Rett Syndrome patients – a rare neurological disorder.
Based near Milan (Italy), Newron is a Biopharma developing therapies for diseases of the central nervous system (CNS). Last year it raised over €23M to advance its pipeline, which features candidates for Parkinson’s disease and schizophrenia.
Now, Newron has received a positive answer from the FDA for its Investigational New Drug (IND) application regarding sarizotan. This therapy is a novel compound for Rett syndrome, which had already been granted an Orphan Drug Designation.
Sarizotan could become the first drug available to patients of Rett syndrome, a rare neurodevelopmental disorder. The disease is associated with mutations in a gene important to neuron function (MeCP2).
Rett syndrome mainly affects females, and is quite rare – one in 10,000. Besides impaired cognitive and motor development, people affected often suffer from respiratory problems,