Core Viewpoint - Roche has received approval from the European Commission for a new room-temperature stable tablet formulation of Evrysdi® (risdiplam) for the treatment of spinal muscular atrophy (SMA), enhancing treatment flexibility and convenience for patients [1][2]. Company Overview - Roche is a leading biotechnology company focused on developing innovative medicines and diagnostics, with a strong emphasis on neuroscience and chronic disease management [10][12]. Product Details - The new 5mg Evrysdi tablet can be taken with or without food, does not require refrigeration, and is suitable for individuals aged two years and older who weigh at least 20kg (44 lbs) [1][4]. - Evrysdi is designed to increase and sustain the production of SMN protein, critical for maintaining healthy motor neurons, thereby improving the course of SMA [2][6]. Clinical Development - The approval of the tablet formulation is based on a bioequivalence study demonstrating that it provides the same efficacy and safety as the original oral solution [3][8]. - Over 18,000 individuals with SMA have been treated with Evrysdi globally, highlighting its established efficacy and safety profile [2][7]. Industry Context - SMA is a severe neuromuscular disease affecting approximately one in 10,000 babies, leading to significant muscle weakness and potential fatality [9]. - Roche's commitment to neuroscience includes investigating multiple treatments for various neurological disorders, including SMA [11].

Core Viewpoint - Roche has received approval from the European Commission for a new room-temperature stable tablet formulation of Evrysdi® (risdiplam) for the treatment of spinal muscular atrophy (SMA), enhancing treatment options for patients [1][2][3]. Company Overview - Roche is a leading biotechnology company focused on developing innovative medicines and diagnostics, with a strong emphasis on neuroscience and chronic disease management [10][12]. - The company has been involved in the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics [4][7]. Product Details - The new 5mg Evrysdi tablet can be taken with or without food, does not require refrigeration, and is suitable for individuals aged two years and older who weigh at least 20kg (44 lbs) [1][4]. - Evrysdi is designed to increase and sustain the production of SMN protein, which is critical for maintaining healthy motor neurons [5][6]. Clinical Efficacy - Over 18,000 patients have been treated with Evrysdi globally, demonstrating its proven efficacy and safety [2][7]. - The approval of the tablet formulation is based on a bioequivalence study that confirmed it provides the same efficacy and safety as the original oral solution [3][8]. Market Impact - The introduction of the tablet formulation is expected to simplify disease management for SMA patients, offering greater portability and convenience [2][3]. - Evrysdi is currently approved in more than 100 countries, indicating its widespread acceptance and potential market reach [7].