Pharmaceutical company Novartis gene therapy for babies – by winning a lottery ticket.
Status: 03.02.2020 3:05 p.m..
The pharmaceutical company Novartis is giving away a gene therapy for 100 terminally ill babies. One dose of the drug costs over two million dollars. Critics speak of a survival lottery.
By Ulrike Till, SWR.
Every few weeks, the Basel pharmaceutical company Novartis wants to hold a raffle: the name of a baby is drawn on behalf of Novartis. A toddler suffering from spinal muscular atrophy will then receive the most expensive drug in the world for free. A Novartis subsidiary has developed drug gene therapy.
The News drug Zolgensma is not yet approved in Europe.
The drug is said to save young children with spinal muscular atrophy. Around one in 10,000 newborns is affected. If left untreated, the disease often ends fatally before the age of two.
Of course, those affected are happy when there is an effective therapy against it – even if it costs over two million.
Novartis wants to give away a drug.
Expensive therapy is just the tip of the iceberg.
The price spiral for very special and rarely requested drugs has been spiraling into alarming heights for a while: The new drug Zolgensma is only the tip of the iceberg.
The drug Luxturna for a rare form of hereditary blindness already costs $ 850,000. And for Kymriah, a tailor-made gene therapy for a subtype of leukemia, health insurance companies have to pay $ 475,000. However, the treatment should work after a single infusion.
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The promise: cure from a single dose.
You also have to consider that if you consider the high prices of new active ingredients to be completely excessive. If a cure is possible with a single dose, it can even save the health system money: because there is no need for years of therapy with equally expensive but less successful agents. With this argument, Novartis also convinced the initially skeptical US drug agency FDA.
But no one knows yet whether the hope of healing will be fulfilled. The first test subjects were only treated five years ago.