Novartis has presented new data reinforcing the clinical benefits of Zolgensma® (onasemnogene abeparvovec), the sole one-time gene therapy approved for spinal muscular atrophy (SMA) treatment. The final results from the SMART study emphasize Zolgensma’s safety and efficacy in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, many of whom (87.5%) had discontinued another disease-modifying therapy before treatment. These findings complement real-world experiences, indicating the therapy’s effectiveness in older and heavier children in countries where age restrictions are not in place.
Dr. Hugh McMillan, a Pediatric Neurologist, highlighted the study’s evidence supporting Zolgensma’s clinical benefits for older and heavier SMA patients, even those previously treated with other therapies. The study aimed to assess Zolgensma’s safety in a broader patient population, with most experiencing manageable increases in transaminases and transient thrombocytopenia. No new safety concerns were identified.
Patients in the SMART study generally maintained motor milestones observed at baseline throughout the one-year study, with positive changes in the Revised Upper Limb Module (RULM) and Hammersmith Functional Motor Scale – Expanded (HFMSE) scores. Four patients achieved new developmental milestones by week 52.
Dr. Sandra P. Reyna, Chief Scientific Advisor at Novartis, emphasized the significance of this open-label study, the first to include older, heavier, and previously treated patients. She suggested that these findings should instill confidence in caregivers and healthcare professionals when making informed treatment decisions for the studied patient population.
Zolgensma is the sole approved gene therapy for SMA, addressing the genetic root cause by replacing the non-functional SMN1 gene through a one-time intravenous infusion. With approvals in over 51 countries, it has treated over 3,700 patients globally. Novartis remains committed to exploring Zolgensma’s potential through ongoing clinical development and investigating intrathecal administration of OAV101 for later-onset SMA forms. Spinal muscular atrophy, a rare genetic neuromuscular disease, is a leading cause of infant death, and Zolgensma’s continuous evaluation aims to redefine possibilities for children living with SMA.