Basel, Switzerland — Novartis shares new data on Zolgensma’s safety and efficacy in older and heavier children with Spinal Muscular Atrophy (SMA), showing maintained or improved motor milestones after 52 weeks. Importantly, the study did not identify any new safety concerns with Zolgensma in this patient group.
SMA is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons, in the spinal cord and the part of the brain connected to the spinal cord (brainstem). The loss of motor neurons leads to muscle weakness and atrophy.
SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1), which is critical for the maintenance of motor neurons. The severity of SMA and the age at which it begins vary significantly among individuals. SMA is typically divided into several types based on the age of onset and the highest physical milestones achieved.
The treatment for SMA has advanced significantly in recent years, with the development of gene therapies like Zolgensma and other treatments aimed at increasing the production of the SMN protein or supporting the function of the nerves and muscles. Results indicate benefits for older patients, with no new safety concerns observed. Zolgensma is a one-time gene therapy for SMA, addressing the genetic cause.
This study aimed to assess the safety and tolerability of Zolgensma, a gene therapy, in patients who are older and heavier than those in prior studies. The main findings revealed that most patients had elevated liver enzymes (transaminases) and temporary low platelet counts (thrombocytopenia).
These side effects were symptomless and effectively managed through monitoring and treatment as recommended in the product’s guidelines.
The SMART study includes patients aged 1.5 – 9.1 years, with most switching to Zolgensma from other therapies.