New four year data for Roche’s Evrysdi reinforce long-term efficacy and safety profile in some of the most severely affected people with types 2 and 3 spinal muscular atrophy (SMA)

• Data from pivotal SUNFISH study showed increases in motor function observed during the first year were maintained through the fourth year, while the overall rate of adverse events continued to decrease
• Data confirm long-term efficacy and safety profile of Evrysdi in a broad range of people with Type 2 and non‑ambulant Type 3 SMA
• More than 8,500 people—from newborns to the over 60s—have been treated with Evrysdi, which is now approved in more than 90 countries worldwide
  
  

Basel, 20th March 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new long-term data for Evrysdi (risdiplam) in a broad range of people aged 2-25 years with spinal muscular atrophy (SMA) from the pivotal SUNFISH study¹. Data confirm increases in motor function were sustained at four years and the overall rate of adverse events continued to decrease over the 48 month period, reinforcing the long-term efficacy and safety of Evrysdi. Participants also reported continuous improvement or stabilisation when independently performing activities of daily living such as eating, drinking and picking up and moving objects. The data were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 19-22, 2023.

“Preserving long-term independence and the ability to perform daily tasks is an important measure for people living with SMA and their caregivers. It’s encouraging to see that Evrysdi has meaningfully affected this aspect of their lives,” said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. “This trial included people with Type 2 and 3 SMA, including patients with advanced disease. These latest data make us confident that the improvement observed during the first year of treatment is sustained during four years which contrasts with the decline we would observe in the absence of treatment.”

The increase in motor function from baseline observed during the first year of the study was maintained through the fourth year of treatment with Evrysdi, as measured by changes in Motor Function Measure 32 (MFM-32) and Revised Upper Limb Module (RULM). Without treatment, natural history data show that patients with Type 2 or 3 SMA typically show a decline in motor function over time. Evrysdi was well-tolerated over the four-year time period. Adverse events (AEs) and serious adverse events (SAEs) were reflective of the underlying disease. The most commonly reported AEs include headache, fever (pyrexia) and upper respiratory tract infection. No treatment-related AEs led to withdrawal from the study.