This is the “most expensive medicine in the world”

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On Friday, the US FDA gave the green light to Zolgensma, which is already known as ” the most expensive drug in the world .” And it is not for less since its final price has been set at $2,125,000.

However, if we look only at the price we will be forgetting the most interesting of 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 in all the world.

A medication for SMA . The Spinal Muscular Atrophy is an autosomal recessive hereditary neuromuscular disease (ie, both parents must be carriers of the gene responsible for the disease).

It affects approximately one in 6,000-10,000 people and manifests as a progressive loss of muscle strength caused by the involvement of the motor neurons of the spinal cord which prevents nerve signals from being transmitted correctly to the muscles (with the consequent atrophy ).

It is not the only medication that exists to treat SMA. Spinraza in Spain was controversial also a few years ago for its high price. However, the Zolgensma of Novartis goes a step further because it is presented as the first great gene therapy available to the general public. A therapy that can cure the disease with a single application.

The medicine that comes. 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 hemophilia or muscular dystrophy await their turn in the face of approval by the highest American authority. Although not only to the approval, of course.

Because the arrival of the Zolgensma opens many debates, especially regarding the financing of these medicines.

Novartis has already begun negotiations with US insurers to allow payment plans and try to introduce these types of therapies in the clinic at prohibitively expensive prices. Above all, because as has happened again, the reasonable thing is to think that in the coming years the price of these treatments will go down a lot.

Patients, pharmaceutics 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 address it with all the guarantees of transparency and fairness.

Last week, the BMJ published a report questioning the close relationship between pharmaceutical companies and patient associations in the United Kingdom. That gene medicine becomes mainstream is great news, but (as we see) in the next few years all the health systems of the world will be put to the test.

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