SMA Newsroom

May 24, 2019

AveXis receives FDA approval for Zolgensma®, the first gene therapy for paediatric patients with SMA

It was announced today that AveXis received FDA approval for Zolgensma®, the first gene therapy for paediatric patients with SMA.

Zolgensma has been approved in the United States for the treatment of patients younger than 2 years of age who have been diagnosed with SMA and including those who were pre-symptomatic at diagnosis. This treatment is designed to address the genetic root cause of SMA by providing a fully functional copy of the defective SMN1 gene in order to, effectively halt disease progression with a single, one-time infusion.

This treatment was pioneered by Dr Martine Barkats, from the Institut de Myologie in France. Funded by AFM-Téléthon and SMA Europe, she successfully treated SMA mice with an AAV9 viral vector.

SMA Europe is delighted with this news as there are now two approved treatments for SMA. Members of SMA Europe have been working hard with AveXis to ensure the patient voice was taken into account whilst the drug was in development. It now anxiously awaits a decision from the European Medicines Agency (EMA) and further development of the intrathecal formulation to address the needs of older patients.

Access of European patients to treatments will continue to be at the top of SMA Europe’s agenda. We acknowledge there is still a long road to walk before European patients can access this new treatment. SMA Europe would regard it as unethical if the price would preclude any patient in any country from receiving this potentially life saving therapy.

SMA Europe will continue working to help health authorities to make informed decisions, that include the patient’s perspective. At the same time, we expect AveXis to commercialise ZOLGENSMA® in Europe putting the patient at the centre of their pricing decisions.