► What treatment is this?
Zolgensma is a drug that treats spinal muscular atrophy. This rare genetic disorder, which affects around 120 births a year in France, causes degeneration of the motor neurons, the nerve cells in the spinal cord that control muscle activity.
Treatment is to replace the mutated gene. It was developed by the American biotech Avexis, which has since been purchased by the Swiss laboratory Novartis. Preclinical research had previously been carried out in France by Généthon, recipient of donations from the Telethon, which signed a license agreement with Avexis.
In May 2019, Zolgensma obtained marketing authorization in the United States, for children under the age of two, for $ 2.1 million (€ 1.86 million). An amount that the laboratory justifies by the cost of development and the effectiveness of the treatment. The Zolgensma dossier is currently being examined by the European and Japanese drug agencies.
► Where did this idea for the draw come from?
The controversies raised by the price of the treatment were rekindled by the participatory prize pools opened by families in different European countries, notably in Belgium where, over a weekend, the parents of a sick child raised the money. It is in this context that the laboratory announced that it will make a hundred doses available free of charge in 2020, to make the drug accessible to families who did not necessarily have the means to buy it.
But for Marilise Marcantonio, director of communications for Novartis, another consideration led to this process which she refuses to call “draw”. “In this extremely serious disease, time is an essential factor and we know that treatment is much more effective before the age of two., she explains. However, the marketing authorizations are long and the level of production complex. We therefore decided to push our production capacity to the maximum to be able to benefit patients, beyond the American market.. “
Doctors are therefore invited to identify cases in their countries and to transmit their files to a platform where they will then be checked and drawn by lot under the supervision of an independent committee. An effort estimated at 200 million euros and received as a provocation by associations.
“How can you imagine telling a family facing the fatal illness of their baby and eligible for treatment, that they are going to be drawn? It’s appalling, unacceptable, just unethical, ” fulminates Christian Cottet, Director General of AFM Telethon and himself the father of a young woman suffering from the disease.
► Why is France not concerned?
All countries are eligible to participate in this draw, with the exception of the United States and France. The United States since the treatment is already authorized and marketed there. France, for different reasons. The treatment is still being examined at European level. If it obtains a marketing authorization, it will still have to be assessed in France and in the Member States, each also fixing its price.
However, in France there is a derogation system of authorization which a clinician can activate if he considers that there is no alternative. These “temporary user authorizations” are covered by social security. Seven babies are already treated by Zolgensma in France today. These are all the more rare since there is already another treatment for spinal muscular atrophy, Spinraza, which is certainly more invasive and restrictive but also effective.
For AFM Telethon, which expressed it in a press release, which is now the most important “Is that Novartis / Avexis is accelerating regulatory procedures to obtain as soon as possible a marketing authorization for Europe (announced at the end of the first quarter of 2020) and accelerating price negotiations in France, by returning to a more reasonable position than the american price “.
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