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Muscular Dystrophy Association (MDA) | 2024 | iPharmaCenter

Novartis presented Zolgensma data demonstrating efficacy in children with SMA weighing between ≥ 8.5 kg to ≤ 21 kg.

Novartis has unveiled fresh findings that reinforce the therapeutic advantages of Zolgensma (onasemnogene abeparvovec), the sole one-time gene therapy authorized for addressing spinal muscular atrophy (SMA).

Conclusive data from the SMART study underscore the safety and effectiveness of Zolgensma in youngsters with SMA, weighing between ≥ 8.5 kg to ≤ 21 kg. The mean age of participants was approximately 4.69 years, with the majority (87.5%) having discontinued another disease-modifying therapy before treatment initiation.

These novel clinical insights complement the burgeoning real-world utilization and experience of this groundbreaking therapy among older and heavier pediatric patients in regions where its use isn't age-restricted.

The principal aim of the study was to assess the safety and tolerance of Zolgensma in a cohort older and bulkier than those enrolled in preceding clinical investigations. Most patients experienced elevated transaminases and transient thrombocytopenia; however, all instances were devoid of symptoms and adeptly managed through vigilant monitoring and intervention, as delineated in the product instructions. No novel safety concerns emerged during the study period.

A significant proportion of patients in the SMART study upheld their motor milestones observed at baseline over the course of the one-year study duration. There was a mean augmentation of 2 points in the total Revised Upper Limb Module (RULM) score and 3.7 points in the total Hammersmith Functional Motor Scale – Expanded (HFMSE) score. Four patients demonstrated the attainment of new developmental milestones by week 52.

SMART constituted a Phase 3b, open-label, single-arm, multicenter investigation devised to evaluate the safety, tolerability, and efficacy of a solitary intravenous infusion of Zolgensma in pediatric patients afflicted with symptomatic SMA possessing bi-allelic mutations in the SMN1 gene, any copy number of the SMN2 gene, and weighing between ≥ 8.5 kg and ≤ 21 kg. The trial encompassed 24 patients with heterogeneous SMA phenotypes across three weight categories, spanning an age range of approximately 18 months to 9 years (mean age of 4.69 years).

Among the participants, three were treatment-naïve to preceding SMN-dependent therapies, while 21 had prior treatment exposure and discontinued either risdiplam or nusinersen before enrolling in the study. Notably, the SMART study marked the inaugural open-label clinical investigation of Zolgensma inclusive of previously treated patients.

Elevated transaminases (ALT or AST >3xULN) were observed in the majority of patients (87.5%), alongside transient thrombocytopenia detected in 70.8% of patients; however, all occurrences remained asymptomatic and were effectively managed. Notably, there were no documented incidents of acute liver failure or bilirubin elevations.

Increases from baseline were observed in RULM and HFMSE scores at Week 52, indicative of functional improvement. Additionally, most patients either sustained or improved their motor milestones from baseline until the conclusion of the study.

Zolgensma (onasemnogene abeparvovec) is an approved gene therapy for SMA, tailored to rectify the genetic root cause of the disease by supplanting the function of the deficient or malfunctioning SMN1 gene, thereby arresting disease progression via sustained SMN protein expression following a single intravenous infusion.

With approvals spanning over 51 nations and more than 3,700 patients globally treated with Zolgensma across diverse clinical trials, managed access programs, and commercial deployments, Novartis remains resolute in its commitment to revolutionize the prospects for children grappling with SMA. The company persists in assessing

Zolgensma's efficacy through an extensive clinical development initiative, alongside the exploration of intrathecal administration of OAV101 in individuals with later-onset SMA forms.


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