Newron Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its new chemical entity (NCE) sarizotan to treat Rett syndrome, a severe neurodevelopmental disorder.
Rett syndrome primarily affects females with an estimated prevalence ranging from one in 10,000 to 20,000 females and there are no approved treatments available.
In preclinical testing with both acute and chronic dosing, Sarizotan, a 5HT1A agonist and D2 agonist/antagonist, has been associated with a 70 to 85% reduction of apneas and hyperventilation episodes.
Preclinical studies have showed that sarizotan has been fully characterized in evaluating its toxicological effects and metabolic profile, without any significant safety concerns.
Recently, the company has also received a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) for sarizotan.
Newron chief medical officer Ravi Anand said: "Newron believes that sarizotan could demonstrate an improvement in key symptoms of Rett syndrome, including episodes of apnea, hyperventilation and breath-holds.
"A reduction in respiratory symptoms is likely to improve quality of life of patients, caregivers, and in the long term, we expect that treatment with sarizotan may reduce secondary cardio-respiratory complications and extend the lives of girls and women with Rett syndrome.
"Newron is currently in advanced discussions with regulatory authorities in Europe, the United States and Canada on the proposed clinical development program."