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FDA approved a new gene therapy treatment in Spinal Muscular Atrophy

The US Food and Drug Administration (FDA) has approved on 24 May 2019 Zolgensma commercialization, the first gene therapy treatment indicated in Spinal Muscular Atrophy type 1. The Swiss laboratory Novartis is waiting for the approval of the European and Japanese authorities current 2019.

Zolgensma is indicated for the treatment of children less than two years of age with Spinal Muscular Atrophy (SMA). This genetic disease involves motor neurons, essential for signal transmission in the whole body to control muscular movements. In the type 1 SMA, the SMN1 gene encoding the SMN protein is defective or absent, causing motoneurons degeneration, leading to a defect in movement order. In newborns with SMA, symptoms appear in the first 6 months of life, which are characterized by muscle weakness in essential functions of life, such as breathing, swallowing, speech or walking, evolving into paralysis or death.

Zolgensma, an innovative gene therapy treatment, is a single intravenous injection of an adeno-associated virus vector (otherwise known as AAV vector), widely used for gene transfer in gene therapy. This vector delivers the functional SNM1 gene into motoneurons, thereby restoring the motor signal and muscular functions.

Developed by the American laboratory AveXis, then bought in May by the Swiss laboratory Novartis, Zolgensma is the most expensive drug ever approved. It price? $ 2.125 million (€ 1.896 million). According to AveXis, this price is explained by its one-time and sustainable administration, unlike it competitor, Spinraza, marketed in 2016 by the Biogen laboratory, which requires 4 injections the first two months of treatment and an injection every 4 months throughout the patient’s life. According to Vas Narasimhan, CEO of Novartis, the cost-effectiveness ratio of Zolgensma is within the range of traditional thresholds used by the Institute for Clinical and Economic Review (ICER). In addition, Zolgensma is expected to save costs in the healthcare system compared to other chronic treatments, such as Spinraza, which can often exceed $ 4 million in just the first 10 years of a young child’s life.

In order to facilitate access to this next-generation treatment, the Novartis laboratory plans floated alternative payment models, by offering insurers ability to pay $ 425,000 a year for five years, and also money-back arrangements if the drug does not work. “We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time” says Vas Narasimhan.

With around 30 new cases per month in the US and 700 eligible patients, this innovative gene therapy drug offers new hope to patients whose “now have another treatment option to minimize the progression of SMA and improve survival”, such as says Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. After the United States, Zolgensma could be marketed in Europe, since Novartis is waiting for the approval of the European and Japanese authorities current 2019.

Aurélie Laubier, PhD, Project manager DIM Thérapie Génique



Novartis press release

FDA press release

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