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Novartis shares new data for Zolgensma in children with SMA

Betsy Goodfellow | March 5, 2024 | News story | |  Musculo-skeletal disorder, Novartis, Zolgensma, sma, spinal muscular atrophy 

Novartis has announced new data to continue the support of the clinical benefits of Zolgensma (onasmenogene abeparvovec), for the treatment of spinal muscular atrophy (SMA).

The data from the SMART study demonstrates the safety and efficacy profile of the drug in paediatric patients with SMA weighing between 8.5kg and 21kg, with a mean age of 4.69 years.

The aim of the SMART trial was to assess the drug’s safety and efficacy in older and heavier patients than previous trials, with most patients included in the trial experiencing increases in transaminases and transient thrombocytopenia.

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The majority of patients in the trial maintained motor milestones observed at baseline throughout the one-year duration of the study. The study saw a mean increase of two points in the total Revised Upper Limb Module (RULM) score, and 3.7 points in the total Hammersmith Functional Motor Scare – Expanded (HFMSE).

Dr Sandra P Reyna, chief scientific advisor and head of global medical engagement for SMA at Novartis, commented: “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.”

Betsy Goodfellow

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