A recent study presented at the Cure SMA Research & Clinical Care Meeting has revealed the sustained efficacy and safety of Evrysdi (risdiplam) in children with Type 1 spinal muscular atrophy (SMA) over a 5-year period.
The key findings demonstrate that:
• By the end of year 5, 91% of treated children remained alive, significantly higher than the typical life expectancy of 2 years without treatment.
• 81% were alive without permanent ventilation, indicating improved respiratory function.
• The majority, approximately 59%, could sit without support for at least 30 seconds.
Furthermore, 96% retained the ability to swallow, and 80% could feed without a feeding tube, indicating preserved feeding and swallowing capabilities. These improvements are crucial for daily living and overall well-being.
Professor Giovanni Baranello, a leading expert in SMA, indicated that the long-term findings confirm the continued benefits of Evrysdi for children with Type 1 SMA.
Motor skills, such as sitting, standing, and walking, were maintained or improved in treated children. Notably, some even demonstrated the ability to stand or walk with support.
The study also reported a decline in overall adverse events by 66% over the 5-year period. Upper respiratory tract infection, pyrexia, and pneumonia were the most common adverse events.
Dr. Levi Garraway, Genentech’s chief medical officer, emphasized the significance of the findings, “This study reinforces Evrysdi’s role as a crucial treatment option, improving the lives of children with SMA worldwide.”
Evrysdi has received approval in over 100 countries, with over 15,000 patients treated globally, demonstrating its widespread reach and impact on the lives of those affected by Type 1 SMA.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
