FDA grants RPDD to ReviR’s RTX-117 for CMT

ReviR Therapeutics has received a RPDD from the US FDA for RTX-117, its investigational oral small-molecule therapy for CMT. The designation covers CMT as a whole and is not restricted to specific subtypes. CMT consists of a group of inherited neuromuscular disorders, often presenting in childhood or adolescence. Credit: SkazovD/Shutterstock.com. ReviR Therapeutics has received a rare paediatric disease designation (RPDD) from the US Food and Drug Administration (FDA) for RTX-117, its investigational oral small-molecule therapy for Charcot-Marie-Tooth disease (CMT). The designation covers CMT as a whole and is not restricted to specific subtypes. LOA and PTSR Model ARGX-117 in…

Source: Pharmaceutical Technology