Evrysdi (risdiplam)

Evrysdi is an approved treatment for spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Evrysdi is a survival motor neuron-2 (SMN2) mRNA splicing modifier. A liquid medicine, it is administered daily by mouth or feeding tube and is the first and only medicine for SMA that can be taken at home. It works to increase and sustain functional survival motor neuron (SMN) protein levels in the central nervous system and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons, responsible for transmitting movement signals from the brain to the muscles. People affected by SMA are dependent on the SMN2 gene to produce SMN protein, as they carry a mutation in chromosome 5q resulting in the SMN1 gene producing insufficient levels of functional SMN protein. Evidence shows that increasing the levels of SMN protein has significant clinical benefits for people living with a wide range of SMA types. The development of Evrysdi is part of a collaboration between Roche, PTC Therapeutics and the SMA Foundation. Evrysdi received U.S. Food and Drug Administration (FDA) approval in August 2020, which was based on data from two clinical studies: FIREFISH and SUNFISH. It is currently licensed in the U.S. only and remains an investigational treatment in all other jurisdictions.