Swiss pharmaceutical company Novartis has reported new findings from the Phase IIIb SMART clinical trial, affirming the safety and efficacy of Zolgensma (onasemnogene abeparvovec) for treating spinal muscular atrophy (SMA).
The multicentre, open-label trial assessed intravenous doses of the one-time gene therapy Zolgensma in paediatric subjects with symptomatic SMA with bi-allelic mutations in the SMN1 gene and varying copy numbers of the SMN2 gene.
The study included participants weighing between 8.5kg and 21kg, aged approximately from 18 months to nine years.
Trial data demonstrated Zolgensma's safety and efficacy profile in these children, most of whom had ceased using other disease-modifying therapies at treatment initiation.
The majority of subjects experienced asymptomatic increases in transaminases and transient thrombocytopaenia, effectively managed according to product guidelines.
No new safety concerns were identified during the trial and most participants maintained or improved their motor milestones over the one-year study period.
The trial also reported a mean rise of two points in the Revised Upper Limb Module (RULM) score and a 3.7-point mean increase in the Hammersmith Functional Motor Scale – Expanded (HFMSE) score.
Four of the trial subjects developed new motor milestones by week 52.
Zolgensma has received approval in more than 51 countries, with more than 3,700 patients treated worldwide in various settings.
Novartis Global Medical Engagement for SMA chief scientific advisor and head Dr Sandra Reyna said: “This data – the first Zolgensma open-label clinical study to include older and heavier, as well as previously treated, patients – should build confidence among caregivers and healthcare professionals as they make informed treatment decisions, consistent with their local product label, for the studied patient population.
“We remain committed to reimagining possibilities for the SMA community.”
Last month, Novartis cancelled its blood cancer candidate sabatolimab after a pivotal trial of the candidate failed to meet its primary endpoint.
