Zolgensma, a gene therapy used to treat spinal muscular atrophy (SMA), is being hailed as a potential cure for the rare inherited disorder. However, recent outcomes have painted a more nuanced picture, with many patients requiring additional therapies on top of Zolgensma. This raises questions about other high-cost gene therapies coming to market.
Baby Ben Kutschke was diagnosed with SMA at three months old. His parents decided to try Zolgensma, a one-time therapy that replaces the genes needed for muscle control. While Ben was able to hold his head up after treatment, he did not progress further and his doctors recommended another drug. This experience reflects the growing number of SMA patients requiring add-on therapies despite receiving Zolgensma.
Zolgensma, launched by Novartis in 2019 as a potential cure for SMA, was the most expensive drug in the world at the time. It has been given to over 3,000 children globally, with 2022 sales of $1.4 billion. However, Novartis’ data shows that almost one-third of children in an ongoing study required other drugs.
The need for additional therapies complicates the pricing of gene therapies, which are often set at very high costs. The long-term benefits of these therapies are being questioned, as researchers argue that the high prices may not be justified if patients still require other treatments. The Institute for Clinical and Economic Review (ICER) suggested that Zolgensma’s maximum price should be $900,000, less than half its current cost.
The pricing strategy for gene therapies is not unique to Zolgensma. Other gene therapies, such as the first hemophilia gene therapy approved by the US FDA, have been priced in the millions of dollars. While drug manufacturers argue that the long-term benefits justify the high prices, critics argue that poor outcomes and the need for additional therapies question the value of these treatments.
The use of add-on therapies after Zolgensma also poses challenges for health insurers, who typically cover the full cost of the treatment upfront. Managing payment plans over time and tracking patients’ responses to treatment require further real-world data.
Despite the need for additional therapies, many parents of children with SMA still believe that Zolgensma is worth it. The treatment has shown success in cases where it was infused soon after birth. However, the timing of treatment is crucial, and patients with more advanced cases of SMA may not experience the same level of improvement.
In conclusion, while Zolgensma has shown positive results in some cases, the need for additional therapies raises questions about the efficacy and value of high-cost gene therapies. The pricing of these treatments will continue to be a topic of debate as more gene therapies come to market.