SMA gene therapy receives approval from US regulator

Published Date
Beatriz Bustillo Ramirez

The US Food and Drug Administration (FDA) has granted approval to Zolgensma, the first gene therapy for spinal muscular atrophy (SMA).

SMA is caused by a fault in a gene called Survival Motor Neuron 1 (SMN1). This gene carries the information required for the production of an important protein. When there is not enough of this protein, the nerve cells that help control the muscles for moving and breathing become damaged.

Zolgensma addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells. For patients under two years of age, it is given as a single, one-time dose into the bloodstream (intravenously). A different delivery method is being investigated in a clinical trial for older patients, where the drug is injected directly into the spinal cord (intrathecally).

The FDA has approved Zolgensma for the treatment of patients under two years of age who have mutations in both copies of the SMN1 gene. This includes children who have not yet experienced symptoms (presymptomatic).

Although the approval process in Europe is separate to the US, it’s a major step forward that this gene therapy may soon be available in US clinics. This is the first gene therapy to be approved for any neuromuscular condition.

The European Medicines Agency (EMA) is responsible for approving potential treatments in Europe and the UK. Zolgensma is currently being reviewed by the EMA and a decision is expected later this year. If the EMA decision is positive, it will be up to the National Institute of Health and Care Excellence (NICE) and the Scottish Medicines Consortium to decide whether to fund the therapy in the UK. NICE is aware of Zolgensma and has started to consider its assessment process.

Further information

Read Avexis’ press release.

Find out more about gene therapy.

If you have any questions about this news story or any other SMA research, please contact the MDUK Research Line on 020 7803 4813 or email

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