In the case of the controversial draw action of a gene therapy for terminally ill babies as well children in Germany now have a Chance. On Monday, the competent Paul gave-Ehrlich-Institute in Langen, the green light, that children in Germany can be treated in the framework of this programme, with the funds Zolgensma, although it is not approved in Europe yet.
There is no reason to speak to the program, informed the Institute. The draw of the first dose began on Monday.
The Swiss pharmaceutical group Novartis, the gene therapy Zolgensma this year, for a total of 100 infants and young children free of charge in countries where there are still no approval. The therapy is for children under two years of age, suffering from spinal muscular atrophy (SMA). The hereditary disease triggers muscle loss and can in severe cases lead to death if left untreated. It is the most expensive drug in the world, with around two million euros for a one-time dose, to help children with SMA.
Mother from Germany: "So you should not entscheiden" about sick children;
For Margaret Ubber from the Stuttgart area that is not, for the time being in issue. She has two affected children, your son but not for the prize draw signed. "So you should not entscheiden" about sick children;, she says the German press Agency. "This should make Doctors, not arbitrary." It bothers you that Zolgensma’ll be touted in the vertebrae to the draw action, often as a healing miracle cure. "For us Spinraza is a Wundermittel", Ubber says. Spinraza in Germany is the only approved SMA-drug. For their children it was a life-saver.
Ubbers daughter Alexa is almost three. You seats, be able to stand with support and is usually a cheerful child. "Without medication you would not be on the Leben", the mother says. The Small was already after birth, virtually unable to move. You will be treated in its tenth week with the since 2017 approved Spinraza. "It makes Riesenfortschritte", Ubber says.
When she was with her now ten-month-old son Theo who is pregnant, was taken to a genetic testing for SMA in the womb. As it turned out, also had the genetic defect that causes SMA. He got his first injection already with three and a half weeks. "He totally gesund", Ubber says. It is crucial to diagnose the disease, before children had the first symptoms. Therefore, Ubber required, SMA in the routine testing of newborn babies to be able to affected children like her son Theo to help, before the first damage is here.
Allocation according to a random principle as the fairest solution
In Germany, according to the Paul-Ehrlich-Institute affected one in 10,000 children of SMA. The particularly severe Form, according to estimates about 50 children. In some Spinraza not have the desired success, as is the case with Marina mantle’s son, Michael. Her son got like two more children in 2019, about a case of hardship Zolgensma. In the case of mantle, and the insurance came up for it. Her son was making progress, she says. Zolgensma is approved since may of last year in the United States. A single injection is expected to reduce the muscle wasting.
Novartis says it could be produced about the expected orders of Zolgensma addition, this year, only 100 doses in addition. The process was time-consuming. So far, there is only one plant in the U.S. state of Illinois, two more U.S. plants are under construction. The company decided, in consultation with ethicists for the award according to the random principle as the fairest solution. Novartis wants to make about the children who win the raffle, no information, not even about how many families have applied.
How Ubber, many consider the prize draw was skeptical. The therapy should, after medical criteria for those children that need it most, says the Federal Director of the German society for Muscle disorders (DGM), Joachim shoot. Medical ethicists Norbert Paul of the University of Mainz says, the receiver circuit would have to be determined by objective, clear criteria. To him the draw feel like a covert marketing campaign, with the help of Novartis a foothold in the market, and pressure to wool.
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