Intended for children with spinal muscular atrophy, Zolgensma has just obtained the green light from the authorities for marketing in the United States. Worth $2.125 million for a single dose, it is the most expensive drug in the world.
It is to date the most expensive medical treatment ever authorized on the market.
Friday, May 24, the Food and Drug Administration (FDA) gave the green light to the pharmaceutical company Novartis for the marketing in the United States of Zolgensma, a revolutionary treatment against spinal muscular atrophy (SMA), a neuromuscular disease that affects the children under two years old. The price of this drug in a single dose has been set at 2.125 million dollars in the United States, or nearly 1.9 million euros. Its launch on the European and Japanese markets should take place during the course of the year.
A “new era” for patients and their families
Affecting between 6,000 and 10,000 children worldwide each year, spinal muscular atrophy (SMA) is a rare genetic disease. Children who suffer from it cannot sit, trudge or walk like the others. As they grow, their muscles continue to atrophy, they may have trouble breathing, and their vulnerability increases.
For Laurence Tiennot-Herment, president of the AFM-Téléthon, the arrival of a drug like Zolgensma therefore offers immense hope for families. “This is amazing news for patients and their families because it is a terrible disease, which leads to paralysis or death of babies,” she explained to BFM TV. A point of view shared by Thibault Soubielle, president of the association Families SMA France, and therefore the little boy is suffering from infantile spinal muscular atrophy “for families, it is a new era which presents itself after years without exit. “
Based on gene therapy, Zolgensma is for them a “medical revolution”, especially since it requires only one dose. “In France, this treatment could concern a hundred children each year”, estimates Laurence Tiennot-Herment, who now wants the European Medicines Agency to grant early marketing for French patients, “with the agreement of the family, the doctor, the industrialist, and the drug agency (ANSM)”.
The price not yet fixed in France
But what about the exorbitant price of the treatment? In an email addressed to BFMTV, Marilise Marcantonio, communication director of Novartis France, wants to be reassuring by explaining that the laboratory is ready to “discuss with the health authorities to imagine new methods in order to guarantee access to this treatment at all patients who need it.
For the moment, the price of Zolgensma has not been fixed in France. “The price must absolutely not be a barrier for patients to access treatment. The discussion will be between the manufacturer and the Economic Committee for Health Products (CEPS)”, assures Laurence Tiennot-Herment, who says however worried “face this escalation of prices”.
For Thibault Soubielle, on the contrary, “the price is never a fight. There is a common interest between the laboratory and the health authorities in finding the right balance point so that it can work”.
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