Evrysdi (risdiplam), marketed by Genentech, a member of the Roche Group, is an FDA-approved therapy to treat SMA. It is an SMN-enhancing therapy that works by targeting the SMN2 gene. It is a small molecule that causes that gene to make more complete SMN protein.

More About Evrysdi

  • Evrysdi is approved for the treatment of SMA in adults and children 2 months and older.
  • Evrysdi was tested in a number of different groups, from infants to adults. It is also being studied in individuals who have previously received a different SMN-enhancing therapy. Individuals in this trial must first go through a “washout” period—that is, they must stop taking the previous medication and wait for it to leave the body—before enrolling.
  • Evrysdi is a daily oral medication. It can also be given through a g-tube.
  • Evrysdi must be taken for the duration of the individual’s life.
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  • For more information on Evrysdi, please click here.
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History of Evrysdi

Evrysdi is an oral medicine that is systemically distributed and designed to increase SMN protein levels in the central nervous system (CNS) and throughout the body. It is designed to help the SMN2 gene produce more functional SMN protein, to better support motor neurons and muscle function. Roche and Genentech led the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics. The approval of Evrysdi was based on four multicenter trials in people with SMA.

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Genentech, a member of the Roche Group, logo