Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 
(SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics 
Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The 
small molecule is designed to treat spinal muscular atrophy caused by mutations 
in chromosome 5q leading to SMN protein deficiency. The drug boosts the ability 
of an alternative gene SMN2 to produce full-length and functional SMN protein. 
In August 2020, Evrysdi™ (risdiplam) received its first approval in the USA for 
the treatment of spinal muscular atrophy in patients 2 months of age and older. 
Risdiplam is in pre-registration for this indication in numerous countries 
worldwide, including the European Union, Brazil, Chile, China, Indonesia, 
Russia, South Korea and Taiwan. This article summarizes the milestones in the 
development of risdiplam leading to this first approval for spinal muscular 
atrophy.