Rett syndrome: Newron Receives FDA Orphan Drug Designation for Sarizotan
Newron Pharmaceuticals S.p.A. announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Newron’s New Chemical Entity (NCE) sarizotan for treatment of Rett Syndrome. Newron also recently announced that it received a positive opinion from the Committee for Orphan Medicinal Products (COMP) from the European Medicines Agency (EMA) for sarizotan.
“Newron believes that sarizotan could demonstrate an improvement in key symptoms of Rett syndrome, including episodes of apnea, hyperventilation and breath-holds,” said Ravi Anand, Chief Medical Officer at Newron. “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 US and Canada on the proposed clinical development program.”
Sarizotan, a 5HT1A agonist and D2 agonist/antagonist, has been associated with a 70 to 85 percent reduction of apneas and hyperventilation episodes in preclinical testing with both acute and chronic dosing. Sarizotan has been fully characterised in preclinical studies evaluating its toxicological effects and metabolic profile, without any significant safety concerns.
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