Roche's Evrysdi Shows Long-Term Efficacy, Safety In Broader Spinal Muscular Atrophy Population

At the annual congress of the Muscular Dystrophy Association (MDA), Roche Holdings AG RHHBY announced new data for its spinal muscular atrophy (SMA) drug Evrysdi (risdiplam).
Evrysdi is a novel survival motor neuron 2 (SMN2) splicing modifier, licensed from PTC Therapeutics Inc PTCT.
At the annual MDA event, Roche’s presentations included new three-year data from the SUNFISH study, which further confirmed the long-term efficacy and safety of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA.
Related: Roche Builds Case For Evrysdi Benefits In Younger Spinal Muscular Atrophy Patients.
There was also exploratory two-year efficacy data from SUNFISH Part 2, demonstrating improvement in or stabilization of motor function with Evrysdi compared to an untreated external control group.
In addition, Roche revealed updated interim data from the RAINBOWFISH study in pre-symptomatic babies with SMA under two months of age.
Price Action: RHHBY shares are down 1.02% at $47.36 during the market session on the last check Wednesday.