This is the "most expensive drug 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 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 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 in everything. the world.

The gene medicine revolution becomes mainstream

Design and architecture

A medicine for SMA. Spinal Muscular Atrophy is an autosomal recessive inherited neuromuscular disease (that is, both parents have to be carriers of 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 medicine that exists to treat SMA. In Spain, for example, Spinraza was controversial also a few years ago due to 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 that comes. According to the FDA, and if all goes well, in 2025 there will be between 10 and 20 gene therapies on the North American market. Treatments for hemophilia or muscular dystrophy await their turn for approval by the highest American authority. Although not only to approval, of course.

Because the arrival of the Zolgensma opens many debates, especially regarding the financing of these drugs. Novartis has already started negotiations with US insurers to allow payment plans and try to introduce this type of therapy to the clinic at prohibitively expensive prices. Above all, because as has happened again, it is reasonable to think that in the coming years the price of these treatments will drop a lot.

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 tackle it with all the guarantees of transparency and fairness. Last week the BMJ released a report questioning the close relationships between pharmacists and patient associations in the UK. That gene medicine becomes mainstream is great news, but (as we see) in the coming years all the health systems in the world are going to be tested. We must get down to work.

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