Novartis’ gene therapy Zolgensma has shown benefit for presymptomatic spinal muscular atrophy (SMA) patients, according to new data presented at the annual European Academy for Neurology (EAN) virtual congress.
The Phase III SPR1NT study was designed to evaluate the safety and efficacy of a one-time intravenous (IV) infusion of Zolgensma (onasemnogene abeparvovec) in presymptomatic children with a genetic diagnosis of SMA, and two or three copies of SMN2.
All patients in the study achieved the primary endpoint of sitting independently for 30 seconds, with 79% of participants achieving this milestone with the World Health Organization (WHO) window of normal development.
In addition, 100% of patients met the secondary endpoint of survival without ventilatory support of any kind at 14 months of age, versus 26% of patients in the Pediatric Neuromuscular Clinical Research (PNCR) natural history cohort.
On top of that, 79% of patients could stand independently, 64% could walk independently and 100% of patients were independent of nutritional and respiratory support for the duration of the study.
In the second Phase III STR1VE-EU trial, Zolgensma demonstrated improvements in motor function and the majority of patients achieved motor milestones not observed in the natural history of SMA Type 1.
“With more than 1,200 children now treated, [this] data presented at EAN further reinforce[s] the life-changing benefit of a one-time treatment of Zolgensma. When treated with Zolgensma prior to the onset of symptoms, not only did all patients survive, but were thriving — breathing and eating on their own and sitting independently, with many standing and walking,” said Shephard Mpofu, chief medical officer, Novartis Gene Therapies.
“When you consider these newborns would go on to develop severe symptoms of SMA Type 1, a devastating, progressive disease that robs children of the ability to talk, eat, sit up and even breathe, findings from the SPR1NT trial are nothing short of extraordinary,” he added.