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Newron Receives Positive Opinion for Orphan Medicinal Product Designation for Sarizotan to Treat Patients with Rett Syndrome from the Committee for Orphan Medicinal Products from the European Medicines AgencyNewron Pharmaceuticals S.p.A. ("Newron"), a research and development company focused on novel CNS and pain therapies, announces that it has received a positive opinion for its New Chemical Entity Sarizotan for the treatment of Rett Syndrome (RTT) from the Committee for Orphan Medicinal Products (COMP) from the European Medicines Agency (EMA (News - Alert)). An application for Orphan Designation has also been filed in the US. Rett Syndrome is a severe neurodevelopmental disorder primarily affecting females with an estimated prevalence ranging from 1:10-20,000 females. There are no approved treatments available. RTT is characterized by a loss of acquired fine and gross motor skills, development of neurological, cognitive and autonomic dysfunction leading to loss of ability to conduct daily life activities, ambulate or communicate. RTT is associated with a reduced life expectancy. Approximately 25% of the deaths in patients with RTT are possibly related to multiple cardio-/respiratory dysrhythmias that result from brain stem immaturity and autonomic failure. More than 95% of the patients present with a mutation in the MeCP2 gene. Prevalence of episodes of apnea, hyperventilation, disordered breathing, etc. are found in approximately 70% of the patients at some stage of their life. Sarizotan, a 5HT1A agonist, and D2 agonist/antagonist has been associated with a 70-85% reduction of apneas and hyperventilation episodes in a genetic (MeCP2) knockout model, both after acute and chronic dosing. Sarizotan has been fully characterized in preclinical studies evaluating its toxicological effects and metabolic profile, without any significant safety findings. Ravi Anand, Newron's CMO, stated: "Newron plans to demonstrate the reduction of episodes of apnea and hyperventilation in patients with RTT by Sarizotan. This reduction in respiratory symptoms is likely to improve quality of life of patients, caregivers and in long term treatment may reduce secondary complications and increase longevity. Newron is currently in advanced discussions with regulatory authorities in Europe, the US and Canada on the proposed clinical development program." An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission.
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