Novartis Reveals Promising Gene Therapy Data for Treating Spinal Muscular Atrophy in Older Children

Monday, Novartis AG NVS presented new data for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for the treatment of spinal muscular atrophy (SMA). 

Final data from the SMART Phase 3b study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease-modifying therapy at the time of treatment. 

The primary study objective was to evaluate the safety and tolerability of Zolgensma in older and heavier patients than were treated in previous clinical studies. 

There were no reported cases of acute liver failure or bilirubin elevations.

The mean increase in total Revised Upper Limb Module (RULM) score was 2 points, and the mean increase in total Hammersmith Functional Motor Scale – Expanded (HFMSE) score was 3.7 points. 

Four patients demonstrated new development milestones at week 52.

Most patients maintained or improved motor milestones from baseline at the time of final analysis.

Nearly all (23/24, 95.8%) patients who could sit with slight support still met this milestone at Week 52.

Three patients achieved the milestone of newly standing with support, and one achieved newly walking with support at Week 52.

All patients (6/6, 100%) who could walk at baseline maintained this milestone until the end of the study.

Most patients in the study experienced increases in transaminases and transient thrombocytopenia; all cases were asymptomatic and managed with appropriate monitoring and treatment, as described in the product labeling. No new safety signals were observed in the study.

In fiscal year 2023, Novartis reported Zolgensma sales of $1.21 billion.

Zolgensma (onasemnogene abeparvovec) is the only approved gene therapy for spinal muscular atrophy. 

The therapy replaces the function of the missing or non-working SMN1 gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. 

Zolgensma is now approved in more than 51 countries, and more than 3,700 patients have been treated with Zolgensma globally across clinical trials.

Price Action: NVS shares are down 0.23% at $101.83 on the last check Monday.

Photo via Wikimedia Commons