Community update from Roche
We are happy to share the latest Community Letter issued by Roche, after the Cure SMA Conference.
Final five-year data from the pivotal FIREFISH (NCT02913482) study were presented by Roche at the Cure SMA Research & Clinical Care Meeting 2024.
Data from the FIREFISH study confirm risdiplam’s long-term efficacy and safety profile in 58 infants with symptomatic Type 1 SMA. After five years of treatment, findings show:
- 91% of children treated with risdiplam were still alive, 81% of whom were alive without permanent ventilation.
- Most children maintained their feeding and swallowing abilities, with 96% able to swallow and 80% able to feed without a feeding tube.
- The majority (59%) of children were able to sit without support for at least 30 seconds.
- The overall rate of adverse events decreased by 66% between year one and the fifth and final year of study. The most common adverse events were upper respiratory tract infection, fever, and pneumonia.
For the full community update, please click below.