New Global Clinical Trial for Onasemnogene Abeparvovec (Zolgensma) Announced

23 April 2021

SMA UK welcomes a new global clinical trial that will be investigating the safety, tolerability and efficacy of intravenous ZolgensmaTM in children who have SMA and weigh between 8.5 kgs and 21 kgs.  

This global trial has an estimated start date of 30th September 2021 and aims to recruit 24 participants in total across sites in Europe, North America, Australia and Taiwan, and will follow patients for a period of 12 months. We understand Novartis is currently exploring the potential of setting up sites in the UK. All currently available information, including inclusion and exclusion criteria, can be found here.

In our submissions to NICE we raised that clinical experts expect that this treatment could also provide health benefits for a proportion of children with SMA Type 2 and 3, and that these health gains would be highly valued by those children and their parents /carers. We acknowledged that no evidence or modelling was presented to NICE to support this and urged Novartis to continue to work to address this gap in access to the treatment. This initiative begins to address this.


Further information

Novartis press release

Novartis Community Statement

About Zolgensma