It costs almost two million euros – the drug, which has so far only been approved in the United States. What is the medicine good for? Can health insurance patients also get it?
The currently most expensive drug in the world can now regularly come onto the market in Germany. Zolgensma received a preliminary EU approval, a spokesman for the EU Commission confirmed on Wednesday. It therefore applies to the treatment of babies and children with a weight of up to 21 kg, who suffer from spinal muscular atrophy (SMA).
Zolgensma is a gene therapy developed by the Novartis subsidiary Avexis. It has been approved in the United States for some time and is priced at $ 2.1 million there. The hereditary disease SMA causes, among other things, muscle wasting. If left untreated, she often leads to death before reaching the age of two.
Zolgensma had recently made headlines because of a raffle by Novartis. Her parents of sick infants and toddlers up to two years old could apply for 100 free treatments. Affected parents, the Society for Muscular Illness and medical ethicists had criticized the raffle. Among other things, they accused the company of a covert marketing campaign and a game with parents’ hopes.
Doctors can prescribe Zolgensma
After approval, Novartis plans to launch the drug in Germany shortly. The exact date is still open, however, said a spokeswoman for the German press agency on Wednesday. After the official market launch, Zolgensma will be able to be prescribed by doctors at the expense of statutory health insurance (GKV), according to the GKV umbrella association.
A spokeswoman for the National Association of Statutory Health Insurance Funds said on request that the price set by the manufacturer would apply in the first twelve months after a drug entered the market. After that, a price negotiated between the manufacturer and the SHI umbrella association will apply.
Why is the drug so expensive?
The basis for the negotiations is a so-called benefit assessment process. Within three months after the marketing authorization of a new drug, it assesses whether an added benefit, if any, is recognized compared to the appropriate comparator therapy.
Novartis company Avexis, responsible for Zolgensma, justifies the high price of its medication by saying that, unlike a competitor’s product, it only needs to be administered once and ensures that higher costs can be avoided. Avexis says that caring for a child with SMA in the first ten years alone costs between 2.5 and four million euros.
The Novartis sucker also pointed out on Wednesday that the company is negotiating flexible pricing models with health insurers. Accordingly, Avexis pays the costs of the therapy up to 100 percent if it does not work.
Zolgensma, according to Avexis, aims to address the genetic cause of spinal muscular atrophy by replacing the function of the missing or non-functioning SMN1 gene. The therapy is administered once intravenously and brings a new working copy of the SMN1 gene into the patient’s cells, which should stop the progression of the disease. According to Avexis, around 500 to 600 children with spinal muscular atrophy are born every year in Europe.
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