Core Viewpoint - Roche has received FDA approval for Evrysdi® (risdiplam) tablet, the only non-invasive disease-modifying treatment for spinal muscular atrophy (SMA), which can be taken whole or dispersed in water [1][2][9] Product Details - Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q, leading to SMN protein deficiency [5][6] - The 5 mg Evrysdi tablet offers the same efficacy and safety as the original oral solution, providing a convenient option for patients [2][9] - The tablet is suitable for individuals aged two years and older who weigh more than 20 kg (44 lbs) [4] Clinical Development and Impact - Evrysdi has been used to treat over 16,000 patients globally and is approved in more than 100 countries [7] - The approval was based on a bioequivalence study showing comparable exposure to risdiplam between the tablet and the oral solution [2] - The new tablet formulation is expected to enhance treatment adherence and simplify disease management for patients and caregivers [3][9] Research and Collaboration - Roche leads the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics [4] - Numerous global multicenter trials are ongoing to evaluate Evrysdi's efficacy and safety across different age groups and SMA types [8][15] Industry Context - SMA is a severe, progressive neuromuscular disease affecting approximately one in 10,000 babies, making effective treatments critical [10] - Roche is committed to advancing neuroscience research, focusing on developing innovative treatments for chronic and debilitating diseases [11][12]
FDA approves Roche's Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA)