Posted by Michael Wonder on 12 Aug 2024
Actio Biosciences receives orphan drug and rare paediatric disease designations for the treatment of Charcot-Marie-Tooth disease 2C
8 August 2024 - Actio Biosciences today announced the US FDA has granted both orphan drug designation and rare paediatric disease designation for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease sub-type 2C.
TRPV4 mutations cause two classes of rare autosomal dominant disorders: peripheral neuropathies including Charcot-Marie-Tooth disease sub-type 2C, and skeletal dysplasias.
