Zolgensma Clinical Trials and Their Results
Zolgensma Clinical Trials and Their Results
Page last checked: 22nd November 2022
The first in-human trial for intravenous (IV) administration of Zolgensma STRIVE began in April 2014. This and the Phase 1 START follow-up trial led to the eventual approval of this one time gene therapy in both the US and Europe for children with SMA Type 1.
In April 2021, a new global clinical trial investigating the safety, tolerability and efficacy of intravenous Zolgensma™ in children (other than those diagnosed with SMA Type 1) who have SMA and weigh between 8.5 kgs and 21 kgs.
In August 2021, the FDA in the US lifted its hold on the clinical trial of intrathecally (IT) administered Zolgensma, allowing Novartis to resume the phase 3 trial called STEER.