Novartis has reported positive data from two Phase III clinical trials which further demonstrated the use of gene therapy, Zolgensma (onasemnogene abeparvovec), for treating spinal muscular atrophy (SMA) in paediatric patients.
Administered as a single intravenous (IV) infusion, Zolgensma is designed to target the genetic root cause of the SMA by swapping the function of the missing or non-working SMN gene to stop disease progression via sustained SMN protein expression.
SMA Type 1 causes progressive and permanent loss of motor function and may lead to death or full-time ventilation by the age of two years, if untreated.
The open-label, single-arm, multi-centre Phase III SPR1NT trial analysed the safety and efficacy of a one-time IV dose of Zolgensma in presymptomatic SMA patients aged six weeks or below with two or three copies of SMN2.
According to results from the two-copy cohort of the Phase III SPR1NT trial, all subjects had age-dependent milestone development, event-free survival and did not need respiratory and nutritional support.
The trial met the primary endpoint with kids sitting independently for 30 seconds or more. Furthermore, 11 of 14 trial subjects could stand independently and nine could walk independently.
No serious, treatment-related adverse events linked to Zolgensma were noted.
The Phase III STR1VE-EU trial assessed the efficacy and safety of a single IV dose of Zolgensma in SMA Type 1 patients aged below six months.
Data from the trial showed that Zolgensma therapy offered quick improvements in motor function.
Nearly 82% of the subjects in this trial attained developmental motor milestones not noticed in the normal history of SMA Type 1. Nearly 49% of them could sit without support for 30 seconds or more.
Novartis Gene Therapies senior vice-president and chief medical officer Shephard Mpofu said: “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.
“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.”
Earlier this month, Novartis reported that its investigational oral drug, iptacopan, met the primary goal in a Phase II trial conducted in patients with IgA nephropathy.