Novartis received a positive CHMP opinion for Itvisma in spinal muscular atrophy patients aged 2 years and older. A European Commission decision is expected within about two months.
Research from Germany and Australia shows zebrafish functional testing can identify false-positive SMA newborn screening results, preventing unnecessary treatment in rare cases where infants carry functional SMN1 gene variants.
Pharmaceutical companies are increasingly partnering with biotechs developing RNA-targeting small molecules, driven by advances in RNA structural biology and the success of drugs like Roche's Evrysdi. The approach aims to address "undruggable" targets while offering oral availability advantages.
Drug discovery is expanding beyond traditional protein targets to include RNA-targeting small molecules, cell surface proteins, and sequence-based AI platforms capable of screening billions of candidates across the entire genome.
