Majority of Children with Severe SMA Maintain or Gain Mobility with Genentech’s Evrysdi: 5-Year Data

Genentech, a member of the Roche Group, shared today groundbreaking 5-year findings reaffirming the lasting effectiveness and safety of Evrysdi® (risdiplam) in children diagnosed with Type 1 spinal muscular atrophy (SMA). These insights stem from the open-label extension of the pivotal FIREFISH study.

By the end of the fifth year, a remarkable 91% of children treated with Evrysdi remained alive, with 81% managing without permanent ventilation. Impressively, the majority could independently sit for at least 30 seconds (59%). Notably, seven children could stand by the study’s conclusion, with three requiring support, four standing unaided, and six walking with assistance. Contrastingly, historical data suggest that without intervention, children with Type 1 SMA rarely live past the age of two, let alone achieve such milestones.

Presented at the Cure SMA Research & Clinical Care Meeting from June 5 to 7, 2024, these long-term results underscore the sustained benefits of Evrysdi for children battling Type 1 SMA. Professor Giovanni Baranello, M.D., Ph.D., from the UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, U.K., emphasized the significant developmental strides observed in children treated with Evrysdi over five years. They not only maintained but often improved their ability to sit, stand, and walk – crucial skills for everyday life. Furthermore, most retained the ability to swallow and eat without a feeding tube.

Assessment of motor function using the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and Hammersmith Infant Neurological Examination 2 (HINE-2) revealed sustained or newfound abilities in those receiving Evrysdi. Additionally, the FIREFISH study demonstrated that most children retained their feeding and swallowing capabilities. By year 5, 96% could swallow, and 80% could feed orally without reliance on a feeding tube.

Dr. Levi Garraway, Chief Medical Officer and Head of Global Product Development at Genentech, hailed the conclusion of the FIREFISH study as instrumental in solidifying Evrysdi’s pivotal role in enhancing the lives of SMA-affected children worldwide. He expressed gratitude to the participants, families, healthcare professionals, and patient support organizations whose unwavering dedication made this progress possible.

Notably, no treatment-related adverse events led to treatment discontinuation or withdrawal from the study, and the overall incidence of adverse events decreased by 66% from year 1 to the study’s culmination. Upper respiratory tract infection, pyrexia, and pneumonia were the most frequently reported adverse events. Furthermore, hospitalizations decreased over the five-year period, with 22% of children not requiring hospitalization since initiating Evrysdi treatment.

Evrysdi stands out as the only oral, non-invasive small molecule SMA therapy designed to target both the central nervous system and peripheral tissues. Genentech spearheads its clinical development through collaboration with the SMA Foundation and PTC Therapeutics.

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