SMA treatment Evrysdi shows multiple benefits for adults
Therapy aids motor function, swallowing, fatigue, life quality
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- Evrysdi stabilizes motor function, improves swallowing, and reduces fatigue for SMA adults.
- It also enhances quality of life for adults with SMA, showing a favorable safety profile.
- Most side effects are mild and temporary, with few discontinuing treatment.
Evrysdi (risdiplam) can stabilize motor function, improve swallowing ability, ease fatigue, and improve life quality in adults with spinal muscular atrophy (SMA), according to a review of scientific literature.
Although the researchers stressed that more studies are needed, they said available data indicate that Evrysdi “shows a favorable safety profile and provides both disease stabilization and multidimensional benefits across all functional [parameters] in adults with SMA.”
The paper, “Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review,” was published in the Journal of Neurology.
SMA is caused mainly by mutations that lead to low levels of the SMN protein, which is key for the survival of motor neurons (the nerve cells that control movement). Lacking SMN, motor neurons sicken and die, which leads to SMA symptoms. Evrysdi is an oral medication designed to boost SMN protein levels.
Evrysdi is widely approved to treat SMA, based on clinical trials showing it can slow disease progression. However, clinical testing of the therapy has primarily focused on children and adolescents, and there’s comparatively little data on Evrysdi’s use in adults with SMA.
Scientists aim for overview of treatment’s effectiveness, safety
Aiming to assess what’s currently known about Evrysdi’s safety and effectiveness in adults with SMA, a trio of scientists in Italy conducted a review of the scientific literature. “This systematic review aims to comprehensively synthesize the available evidence on the safety, effectiveness, and tolerability of [Evrysdi] in adult SMA patients, providing an updated overview of its therapeutic profile in this specific population,” they wrote.
The review encompassed 14 studies, collectively reporting data from more than 200 adults with SMA who started treatment with Evrysdi. Most of the patients had SMA type 2 or type 3; most were unable to walk when they started treatment and had not previously received any other SMA treatment.
Most studies reported on how Evrysdi treatment affected scores on the Revised Upper Limb Module (RULM), a standardized measure of arm and hand function. Across the studies, some adults with SMA showed “modest but clinically relevant improvements” in RULM scores after starting Evrysdi, the researchers said. Patients who didn’t show improvements still tended to have stable RULM scores, which contrasts with typical SMA progression, where motor function worsens over time.
Other standardized measures assessing gross (whole-body) motor function generally showed similar trends. Some patients had modest but notable improvements, and scores overall showed stability while on Evrysdi. The researchers noted that patients who showed improvements tended to be younger and/or have less substantial weakness when they started treatment.
Beyond measures of motor function, studies found that adults with SMA tend to report improvements in swallowing and speaking abilities after treatment with Evrysdi. Patient-reported data also showed that the therapy eased fatigue for most patients and generally led to improvements in quality of life.
“Adults [with SMA taking Evrysdi] described feeling more confident in everyday activities, less frustrated by functional limitations, and more positive in their day-to-day outlook,” the researchers wrote.
Safety data from across the studies showed Evrysdi has a “favorable and generally manageable safety profile” in adults with SMA, the researchers said. Most side effects were mild and temporary, with the most common being digestive problems, skin issues, or elevated liver enzymes (a sign of liver damage). Some patients temporarily paused treatment, but few discontinued it entirely. No deaths or serious safety problems related to Evrysdi were reported in any of the studies.
The researchers stressed that the review was limited to a small number of studies, most of which were fairly short term, so additional studies are needed to evaluate long-term outcomes for adults with SMA who take Evrysdi. Still, they said, the available data indicate that Evrysdi “appears to be safe and generally well tolerated in adults with SMA, and current evidence suggests that it can stabilize disease progression and, in some cases, produce modest but clinically relevant improvements.”
Evrysdi is sold in the U.S. by Genentech, a subsidiary of Roche. Neither Genentech nor Roche was involved in the study.
