Feedback from gene therapy in children with SMA over 8.5 kg

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The therapeutic trials leading to the approval of the onasemnogene abeparvovec (Zolgensma) were conducted in children with SMA weighing less than 8.5 kg. In the framework of a compassionate access programme set up by Novartis for this innovative therapy (GMAP programme), this weight limit could be exceeded:

  • 102 children have been treated since September 2021 for this broader indication;
  • five of them even weighed more than 13.5 kg when injected;
  • for 53% of the children treated, side effects, mostly minor, have been reported;
  • three patients died of respiratory infection, but apparently unrelated to the treatment itself;
  • liver toxicity, thrombocytopenia, vomiting and fever were also transiently reported .

These results confirm a good tolerance of the gene therapy product above 8.5kg.

 

Safety of Onasemnogene Abeparvovec for Patients With Spinal Muscular Atrophy 8.5 kg or Heavier in a Global Managed Access Program. Chand DH, Mitchell S, Sun R et al. Pediatr Neurol. 2022 Jul;132:27-32.

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