Babies with spinal muscular atrophy able to sit up after 1 year of Evrysdi treatment

Key takeaways:

• The study examined 26 newborns with spinal muscular atrophy given Evrysdi.
• Data showed 81% of babies were able to sit without aid for at least 30 seconds after 1 year of treatment.

Genentech has announced positive results from its primary analysis of an ongoing study assessing safety and efficacy of Evrysdi in babies with pre-symptomatic spinal muscular atrophy.

Data from the RAINBOWFISH study were presented at the 28th World Muscle Society Congress, held from Oct. 3 to Oct.7 in South Carolina.

According to a company release, the study met its primary endpoint as 80% of the efficacy population was able to sit upright without support for at least 5 seconds after 1 year of Evrysdi (risdiplam, Genentech) treatment, assessed by Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III).

Per additional data cited in the release, of the 26 babies aged from birth to 6 weeks in the study, 81% could sit independently for 30 seconds, including all patients with low compound muscle action potential amplitude at baseline, with the majority able to stand and walk.

“Evrysdi is the only non-invasive SMA treatment and can be used within hours of birth, potentially allowing these babies to sit, stand and walk, similar to healthy individuals,” Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of Global Product Development, said in the release. “Evrysdi has now demonstrated its safety and efficacy in babies, children and adults, and these compelling data continue to reinforce our confidence in this treatment.’’