New success for Newron Pharmaceuticals. The FDA approved Orphan Drug designation to its new chemical entity Sarizotan, designed to treat patients suffering from Rett syndrome. This decision fits the one from the European Medicine Agency, whose Committee for Orphan Medical Products positively reviewed the drug last month.

The Italian pharma company Newron with a market capitalization of €400M seems entirely recovered from its toughest period, which took place back in 2011. Its lead Parkinson drug Xadago failed at that time in Phase III due to organizational problems and the break down caused the partner Merck to retire from the collaboration concerning this drug.

Newron’s candidate Sarizotan, licensed from Merck, is designed to treat the rare Rett syndrome, a neurodevelopmental disease that leads to impairments in fine and gross motor skills. Preclinical testing demonstrated that the drug led to a 70 to 85% reduction of apneas and hyperventilation episodes, one of the main symptoms of Rett syndrome.

Saratozin’s clinical development program is pushed in the States, Canada and also in Europe, where the candidate received a favorable opinion from the European organization in charge of granting the EU Orphan Medical designation.

The FDA´s designation represents new hope for all patients suffering from Rett syndrome since there is currently no available treatment today. It also embodies good news for Newron, since it will facilitate the performance of clinical trials, reduce their costs and, surely, positively influence the stock market.

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