Novartis Sells the Most Expensive Drug in the World: $5.2 Billion for Only 3,000 Doses

In 2019, Novartis announced the arrival on the market of one of its most anticipated drugs in recent years: Zolgensma. Six years later, it remains the world’s only gene therapy indicated for the treatment of spinal muscular atrophy (SMA), a disease that damages and kills motor neurons.

Beyond the benefits it represents for those affected by this rare disease, Zolgensma has become popular for being the most expensive medicine in the world. Where is it sold, how much does each dose cost, why does it have this price and how much money does it move?

Zolgensma is available in 47 countries

The price per dose varies by territory and ranges between $1.2 million and $2.1 million, according to calculations by the Institute for Clinical and Economic Review (Icer), an independent research institute that produces reports on the effectiveness and the value of the medicines that come to market. Although Novartis has never revealed the exact number of doses sold, in March 2023 it assured that “more than 3,000 patients ” have already benefited from this treatment, so the average price per dose is around $1.7 million.

In Spain, since the end of 2021 it has been financed by the National Health System (SNS). In this case, it agreed with Novartis on a lower price: 1.34 million euros per dose. Even so, Zolgensma became the most expensive medicine in Spain paid for with public money. At the moment, the drug still occupies the throne. According to PlantaDoce calculations, the Novartis drug has already raised $5.2 billion worldwide.

Even so, it is not the company’s drug with the highest turnover: the pharmaceutical company’s annual reports consulted by this medium indicate that, between 2020 and 2023, Zolgensma has oscillated between position nine and thirteen in the ranking of the twenty best-selling drugs in Novartis. Over the last few years, turnover has remained stable, around $1.3 billion annually .

A justified price for Zolgensma?

Despite the high amount per dose, experts assure that it is within reasonable prices . “Zolgensma is dramatically transforming the lives of families affected by this devastating disease and, given the new efficacy data for the pre-symptomatic population, the price is within the price reference range based on the value of Icer,” assessed Steven D. Pearson, president of Icer.

This organization estimates the value of each year of life gained thanks to treatment between $100,000 and $150,000. “Novartis openly talked about charging five million dollars per dose, so it is appreciated that it chose to set a price more within the reach of the families of the affected children,” he highlighted in a report.

According to PlantaDoce calculations, the Novartis drug has already raised $5.2 billion worldwide

Consulted by PlantaDoce, genetic counselor Núria Capdevila explains the high price of the drug compared to the usual costs of gene therapies. “In this type of treatment, it is not just about developing a molecule that works and that manages to successfully pass all clinical trials,” recalls the expert, a genetic advisor at the consultation of the Genetic Council of the Parc Taulí Health Corporation, in Sabadell. (Barcelona).

Capdevila alleges the added costs of the production process. “In order to manufacture and maintain these targeted therapies, ultra-freezers are required that keep the drug at 65 degrees below zero, laminar flow layer hoods, and a clean room in which to generate and maintain them,” she points out.

However, pharmaceutical laboratories are increasingly interested in developing this type of therapies. “Despite the high costs of gene therapies compared to conventional ones, it is evident that they have a huge new market ahead of them and at the moment very little explored,” said the expert. Genetic therapies are those that focus on modifying people’s genes in some way. “The modification of genes, which are our basic units of information, can be applied to solve diseases or reduce the alterations they produce,” she specifies.

Currently, there are very few gene therapies like Zolgensma. Hence also its price. “There are only about ten to treat a very small set of diseases, but it is expected that they will help in the treatment of minority pathologies such as Angelman or Prader Willi syndrome , or the development of common diseases such as diabetes or hypertension,” the expert ventured.

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(Featured image by jniittymaa0 via Pixabay)

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