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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Check out Genentech’s patient brochure on Evrysdi.
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.
Recently Shared News on Evrysdi
- 09/30/2020 – Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA
- 08/11/2020 – Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community
- 08/07/2020 – Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA
- 06/12/2020 – Genentech Announces 2-Year Risdiplam Data From SUNFISH and New Data From JEWELFISH in Infants, Children and Adults with SMA
- 04/28/2020 – Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 SMA
- 04/07/2020 – Community Statement from Genentech on Risdiplam Approval Timeline
- 02/06/2020 – Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA
- 01/23/2020 – Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants with Type 1 SMA
- 11/25/2019 – Community Statement from Genentech: FDA Grants Priority Review to Risdiplam for Spinal Muscular Atrophy
- 11/11/2019 – Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People With Type 2 or 3 SMA
- 10/02/2019 – Risdiplam Data Presented at World Muscle Society Congress Demonstrates Continued Benefit for Patients with SMA
- 05/07/2019 – Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in SMA at the AAN Annual Meeting