Core Viewpoint - Biogen Inc. has received marketing authorization from the European Commission for a high dose regimen of SPINRAZA (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), which is the most common form of the disease, accounting for approximately 95% of all SMA cases [1][2] Product Authorization and Dosing Regimen - The updated SPINRAZA regimen includes 50 mg/5 mL and 28 mg/5 mL doses, featuring a more rapid loading phase with two 50 mg doses administered 14 days apart, followed by 28 mg maintenance doses every four months [1][11] - Individuals transitioning from the previous 12 mg dose will receive one 50 mg dose instead of their next 12 mg dose, followed by the 28 mg maintenance doses [1] Clinical Study and Efficacy - The European Commission's approval is based on data from the Phase 2/3 DEVOTE study, which demonstrated statistically significant improvements in motor function for treatment-naïve, symptomatic infants using the high dose regimen compared to a matched sham group [2][6] - The pivotal cohort showed a mean difference of 26.19 points on the CHOP-INTEND scale, with a significant p-value of <0.0001 [2][9] - Participants transitioning from the low dose regimen also experienced a mean improvement of 1.8 points on the Hammersmith Functional Motor Scale – Expanded from baseline to Day 302 [2] Safety Profile - The high dose regimen was generally well tolerated, with adverse events consistent with SMA and the known safety profile of nusinersen, with no new safety concerns observed [3][6] - Common adverse events reported in at least 10% of participants included pneumonia, COVID-19, pneumonia aspiration, and malnutrition [3] Community and Regulatory Engagement - Biogen is committed to advancing care for individuals living with SMA and is working with regulatory authorities globally to progress the high dose regimen, which is also approved in Japan and under review by the U.S. FDA [7][11] - The approval highlights the importance of sustained research and investment in SMA care, as noted by community leaders [5]

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].