Newron Pharmaceuticals has secured rare paediatric disease designation for sarizotan from the US Food and Drug Administration (FDA) for the treatment of Rett syndrome.
Sarizotan was granted the orphan drug designation from the FDA for the treatment of the rare neurodevelopmental disorder. Rett syndrome is known to affect mainly females and currently has no approved treatments.
The chemical entity, which was licensed by Newron from Merck, is said to be a highly selective compound for specific serotonin or dopamine receptors that regulate the activity of the neurotransmitters in the brain.
As per the FDA, a rare paediatric disease is defined as a serious or life-threatening disease that mainly affects individuals aged 18 years or younger, and impacts less than 200,000 people in the US.
Through the FDA rare paediatric disease designation, drug developers get incentives to advance the development of rare disease drugs and biologics.
Apart from that, the FDA’s rare paediatric disease priority review voucher programme states that a sponsor having a rare paediatric disease designation who gets marketing approval for a rare paediatric disease can be eligible for a voucher that can be redeemed to get priority review for any subsequent marketing application.
Newron chief medical officer Ravi Anand said: “The decision of the FDA to designate sarizotan for the treatment of a rare paediatric 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.
“This designation also represents progress towards qualifying sarizotan for a rare paediatric disease priority review voucher upon potential US marketing approval in the future.”
Currently, Newron is conducting the STARS trial across the US, Europe, Asia and Australia to assess the efficacy, safety and tolerability of sarizotan in Rett syndrome patients who have respiratory symptoms. Results from the STARS trial are expected to be released in the coming few weeks.
Based in Italy, Newron is engaged in developing drugs for diseases of the central and peripheral nervous system. The company has an FDA-approved drug in its portfolio in the form of Xadago/safinamide for the treatment of Parkinson’s disease, which is being commercialised in the US by US WorldMeds.