- At the annual congress of the Muscular Dystrophy Association (MDA), Roche Holdings AG (OTC: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 (NASDAQ: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.
