Posted by Michael Wonder on 26 Feb 2021
Retrotope granted rare paediatric disease designation from FDA for lead development candidate, RT001, in two life-threatening neurodegenerative indications
25 February 2021 - RT001 also granted fast track designation by FDA in Friedreich’s ataxia.
Retrotope, today announced that the U.S. FDA has granted two rare paediatric disease designations to RT001, the company’s lead development candidate.
The first rare paediatric disease designation is for the treatment of infantile neuroaxonal dystrophy, with the second covering the treatment of Friedreich’s ataxia.
