Key findings:
- Significant finding that infants were sitting greater than 5 seconds after a year on treatment.
- A significant improvement in motor function
- children achieved unexpected milestones of sitting or even standing
- majority of children maintained ability to swallow
This is an important stage in the development of information to support a submission for Marketing Authorisation. At present FDA PDUFA has already been submitted in line with the agency’s procedures for ongoing review of critical medicines. The application has also been made in China, Brazil, Chile, Indonesia, Russia, South Korea and Taiwan.
Submission is on track for mid-year EMA now that they have PRIME status. Other country submissions are planned.
We continue to hope for successful review and approval in the EU which we hope will include the UK.
This is an exciting moment as we now have Spinraza approved, there is a positive opinion in the EU for the gene therapy Zolgensma due to be approved in the next few days. Risdiplam is a critical addition to the needed treatments for SMA.
Click here for a summary of information.
