Evrysdi is an approved treatment for spinal muscular atrophy (SMA) in adults, children and infants aged 2 months and older.
Evrysdi is a survival motor neuron-2 (SMN2) mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube, making it the first and only medicine for SMA that can be taken at home. It works to increase and sustain the production of the SMN protein. 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 and was approved by the European Commission in March 2021.
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals and patients are asked to report any suspected adverse reactions. See our information for details on how to report.