Novartis has revealed new data on its one-time gene therapy for spinal muscular atrophy (SMA) in older children.
Nearly all participants in a Phase IIIb trial maintained motor milestones after 52 weeks, according to new data in older, heavier children treated with Novartis' spinal muscular atrophy (SMA) gene therapy or improved.
Final data from Zolgensma's Phase IIIb SMART study® (onasemnogene abeparvovec) highlighted its safety and efficacy in children weighing 8.5 kg to 21 kg and with a mean age of 4.69 years.
The results of this study demonstrated that most children maintained their motor milestones throughout the 1-year study.
According to Novartis, key data highlights include:
- Most patients maintained or improved motor milestones from baseline at final analysis
- 95.8% of patients who were able to sit with minimal support still achieved this milestone at 52 weeks.
- At week 52, three patients achieved the milestone of newly supported standing and one patient was newly able to walk with support.
- All patients (100%) who were able to walk at baseline maintained this milestone until the end of the study.
The importance of new data in Novartis gene therapy
Dr. Sandra Reyna, chief scientific advisor and head of SMA global medical engagement at Novartis, said the data is “the first of its kind to not only look at older, heavier patients, but also patients who have received prior treatment.” This is an open-label clinical study of Zolgensma.
Result is, [Novartis] SMART research provides evidence that: [the gene therapy] Zolgensma is clinically beneficial for older and heavier SMA patients. ”
“The results of the SMART study provide evidence that Zolgensma is clinically beneficial for older, heavier patients with SMA, many of whom have been previously treated with another disease-modifying therapy.” said neurologist Dr. Hugh McMillan. “These data inform the use of Zolgensma in children weighing up to 21 kg and support the use of one-time gene replacement therapy as a treatment option for SMA in the broader population.”
The company also said that overall, “the new clinical results complement new real-world experience,” and that the use of the treatment in older, heavier children “in countries where use is permitted by age.” Shared.
These data are part of the Zolgensma dataset to be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical Science Conference, Novartis confirmed.
This data release follows last year's Novartis study, which demonstrated that gene therapy enabled participants in a long-term follow-up study to achieve 100 percent of all milestones assessed in participants who received Zolgensma before the onset of SMA symptoms. This follows the announcement.