Does Evrysdi cure spinal muscular atrophy (SMA)?

Evrysdi does not cure spinal muscular atrophy (SMA), it just replaces a protein that is low or missing in newborns, children, or adults with the condition, improving muscle strength and allowing some relief from some of the symptoms of SMA. Evrysdi must be taken for a person’s lifetime; if Evrysdi is stopped the newborn, child, or adult’s SMA symptoms will progressively worsen at a faster rate.

How does Evrysdi work?

Evrysdi (risdiplam) works by targeting the SMN2 gene (survival motor neuron gene 2), causing it to make more functional SMN protein. This increases SMN protein levels throughout the central nervous system and body, helping to improve motor nerve and muscle function in children and adults with SMA.

Evrysdi was approved by the FDA on August 7, 2020 and is available as an oral tablet that can be swallowed whole or dispersed in unchlorinated water, or an oral liquid that is given by mouth or through a gastrostomy tube (g-tube). Both formulations are taken once a day after a meal. It may be used to treat all types of spinal muscular atrophy (SMA) in adults, children, and newborns. A label extension for Evrysdi in May, 2022 to include pre-symptomatic infants aged under 2 months (or newborns) allows healthcare providers to intervene as early as possible in treating babies with SMA.