Rare paediatric disease designation for sarizotanJanuary 6, 2020
The US FDA has granted the Rare Pediatric Disease designation for sarizotan for the treatment of Rett syndrome, a rare neurodevelopmental disorder primarily affecting females.
The FDA defines a “rare pediatric 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 US.
The Rare Pediatric Disease designation provides incentives to advance the development of rare disease drugs and biologics.
Newron has completed patient enrollment in the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study, a clinical study to evaluate the efficacy, safety and tolerability of the drug in patients with Rett syndrome suffering from respiratory symptoms.
Among the core symptoms of Rett, breathing disturbances may affect the whole person’s body; they can have a marked effect on biochemistry, influence emotions, circulation and digestive function as well as musculoskeletal structures in the respiratory process.