A real-world study has confirmed the safety and efficacy of Novartis’ gene therapy Zolgensma (ingredient: onasemnogene abeparvovec) in treating pediatric patients with spinal muscular atrophy (SMA) weighing up to 15 kilograms.

The study demonstrated the safety and efficacy of Zolgensma in pediatric SMA patients of all ages. It also showed effectiveness in children under 24 months and those who received Spinraza (ingredient: nusinersen) before.

On Oct. 28, the Lancet Child & Adolescent Health published a real-world study that reaffirmed the safety and efficacy of Novartis’ gene therapy Zolgensma (ingredient: onasemnogene abeparvovec) for pediatric patients with spinal muscular atrophy (SMA).
On Oct. 28, the Lancet Child & Adolescent Health published a real-world study that reaffirmed the safety and efficacy of Novartis’ gene therapy Zolgensma (ingredient: onasemnogene abeparvovec) for pediatric patients with spinal muscular atrophy (SMA).

A research team, led by Professor Claudia Weiss at Charite University Hospital in Berlin, published the results of a cohort study of Zolgensma on SMA patients under two years old and weighing 15 kg or less in the online edition of The Lancet Child & Adolescent Health on Oct. 28.

According to Novartis, the cohort study reaffirmed the safety and efficacy of Zolgensma shown in previous studies, especially expanded safety and efficacy in children after six months of their birth and weighing 8.5 kg or more.

Professor Weiss’ study proved its value due to the rarity of real-world evidence that showed efficacy and safety of Zolgensma in a wide patient group without age restrictions, it said.

The research team conducted a multicenter prospective observational study between September 2019 and April 2021 in 18 pediatric neuromuscular centers in Germany and Austria. Among the 76 SMA patients who participated in the study, 51 were with type 1, 19 were with type 2, and six were presymptomatic. Fifty-eight subjects also had a history of receiving Spinraza, which made the study include the largest number of patients among previous reports that evaluated Zolgensma in patients who used Spinraza.

More than 80 percent of patients who received Zolgensma scored significantly better in the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) and Hammersmith Functional Motor Scale Expanded for SMA (HFMSE) in the six-month follow-up. 

The average CHOP-INTEND of 16 patients younger than eight months increased by 13.8 points, and those between eight months and two years showed a 7.7-point improvement, demonstrating clinically significant improvement in motor function.

In addition, among the 58 patients who had been previously treated with Spinraza, the CHOP-INTEND score increased by an average of 8.8 points in 45 children whose score was confirmed after six months of follow-up.

The research team said the safety profile of Zolgensma reported in the study was consistent with those reported in previous clinical trials and other real-world studies, with no signs of new safety issues reported.

“Considering the diverse patient pools, six-month short observation period, the potential bias of the study, and a history of having Spinraza in the majority of participants, we concluded that Zolgensma to be safe and effective in SMA patients up to 15 kilograms,” the research team said.

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