This is the “most expensive medicine in the world”: the gene medicine revolution is about to become mainstream.

On Friday, the US FDA gave the green light to Zolgensma, which is already known as “the most expensive drug in the world”. And not for less, since its final price has been set at 2,125,000 dollars. However, if we look only at the price we will be forgetting the most interesting thing about this genetic treatment for spinal muscular atrophy: with dozens of therapies waiting to be approved, its short-term future is also that of gene medicine around the world.

The revolution of gene medicine becomes mainstream

A medicine for SMA. Spinal Muscular Atrophy is an autosomal recessive inherited neuromuscular disease (i.e., both parents must carry the gene responsible for the disease). It affects approximately one in 6,000-10,000 people and manifests itself as a progressive loss of muscle strength caused by the involvement of motor neurons in the spinal cord that prevents nerve signals from being properly transmitted to the muscles (with consequent atrophy).

It is not the only drug available to treat SMA. In Spain, for example, Spinraza was also the subject of controversy a few years ago because of its high price. However, Novartis’ Zolgensma goes one step further because it is presented as the first major gene therapy available to the general public. A therapy that can cure the disease with a single application.

The medicine to come. According to the FDA and if all goes well, in 2025 there will be between 10 and 20 gene therapies in the North American market. Treatments for haemophilia or muscular dystrophy await approval by the highest American authority. Although not only to the approval, of course.

Because the arrival of the Zolgensma opens up many debates, especially regarding the financing of these medicines. Novartis has already started negotiations with US insurance companies to allow payment plans and try to introduce this type of therapy in the clinic at prohibitively expensive prices. Above all, because as has happened again it is reasonable to think that in the next few years the price of these treatments will fall dramatically.

Patients, pharmacists and health systems. In Europe, where the public has a much more important role in health systems, the debate is also open, and we must prepare to deal with it with all the guarantees of transparency and fairness. Last week the BMJ published a report calling into question the close relationship between pharmaceuticals and patient associations in the United Kingdom. The fact that gene medicine is becoming mainstream is great news, but (as we see) in the next few years all the world’s health systems are going to be tested. We need to get down to work.