Posted by Michael Wonder on 20 Nov 2019
Newron receives FDA rare paediatric disease designation for sarizotan for the treatment of Rett syndrome
19 November 2019 - Newron announced today that the U.S. FDA has granted the rare paediatric disease designation for sarizotan, the company’s product candidate for the treatment of Rett syndrome, a rare neurodevelopmental disorder primarily affecting females, with no approved treatments currently available.
The U.S. FDA defines a “rare paediatric disease” as a serious or life-threatening disease primarily affecting individuals age 18 years or younger that impacts fewer than 200,000 individuals in the United States.
