Rare Pediatric status for sarizotan in Rett syndrome

The US Food and Drug Administration has granted the Rare Pediatric Disease designation for sarizotan, a product candidate for the treatment of Rett syndrome, a rare neurodevelopmental disorder primarily affecting females, with no approved treatments currently available, under development at Italian drugmaker Newron Pharmaceuticals (SIX: NWRN).

Newron, whose shares rocketed up more than 21% to 7.05 euros on the news today, acquired rights to co-develop sarizotan, along with pruvanserin, with Merck KGaA (MRK:D E) in 2011, but the German firm returned its rights to Newron in 2012.

“The decision of the FDA to designate sarizotan for the treatment of a rare pediatric population, following an earlier decision to grant it an Orphan Drug designation (ODD), highlights the critical need within the Rett community for treatments for this devastating disease,” stated Ravi Anand, Newron’s chief medical officer.