Genentech, a member of the Roche Group, today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Evrysdi (risdiplam) to treat adults and children 2 months and older with spinal muscular atrophy (SMA).

Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA. Infants achieved the ability to sit without support for at least 5 seconds, a key motor milestone not normally seen in the natural course of the disease. Evrysdi also improved survival without permanent ventilation at 12 and 23 months, compared to natural history. A liquid medicine, Evrysdi is administered daily at home by mouth or feeding tube.

The approval of Evrysdi reaffirms the commitment made by the entire SMA community to develop effective treatments for those impacted by the disease. This treatment has the potential to be both lifesaving and to allow for more independent participation in the activities of daily life. We will now begin to work closely with Genentech and Roche, healthcare professionals, and individuals affected by SMA from across the U.S. to ensure rapid access to Evrysdi, according to its FDA-approved label.

Evrysdi is an SMN-enhancing therapy that works by targeting the SMN2 gene. It is a small molecule that works throughout the body to cause that gene to make more complete SMN protein.

Cure SMA and Genentech to Co-Host SMA Community Webinar

Cure SMA and Genentech will hold a joint webinar on the approval of Evrysdi on Tuesday, August 18, 2020 at 12 p.m. (CT). We will be sharing the latest on this news and will include a Q&A session based on questions submitted in advance of the webinar. To register for this webinar and submit a question, please click here. This webinar is intended for U.S. audiences only.

Thank You

Cure SMA would like to extend our deepest gratitude to all the individuals and families, supporters and donors, and partners who have contributed to this milestone. We are especially appreciative of our partners at Genentech, who have worked to develop and implement a clinical testing program that incorporated the broad population of people living with SMA.

Questions? Please reach out to [email protected].