New interim data from Novartis’ phase 3 trial investigating the efficacy of its gene therapy Zolgensma in the treatment of spinal muscular atrophy Type 1 in patients under six months old has revealed that Zolgensma “successfully transduced intended targets in the central nervous system and provided widespread SMN expression comparable to tissue from unaffected individual” following a one-time intravenous infusion of the therapy.
Zolgensma was shown to extend event-free survival in patients as 21 of 22 participants in the trial were alive and event-free as at 27th September 2018, with a median age of 9.5 months. Data indicates that in untreated patients, half of all babies with SMA Type 1 will either die or require permanent ventilation by the age of 10.5 months. There was also a premature and sharp increase in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores, and preliminary evaluations identified there was greater achievement of motor milestones among patients treated with Zolgensma compared with untreated patients. Three patients were able to sit without support for a minimum of 30 seconds as at 27th September 2018, increasing to eight patients by 31st December 2018.
“Positive interim phase 3 trial data for Novartis’ Zolgensma.“
Chief Medical Officer at AveXis, Dr Olga Santiago, said: "These STR1VE data reinforce what was seen in the pivotal Phase 1 START trial, including trends toward prolonged survival and milestone achievement never seen in the natural history of the untreated disease. With a patient population and baseline characteristics closely matched to the START trial, these data build upon the body of evidence supporting the use of Zolgensma for SMA Type 1."