Parents horrified: pharmaceutical company raffles gene therapy for terminally ill babies

Children in Germany now also have a chance in the raffle of a gene therapy for terminally ill babies. The responsible federal institute gave the green light on Monday.

The Swiss pharmaceutical company Novartis started the raffle for a treatment with Zolgensma for 100 children on Monday. It is the most expensive drug in the world, with around two million euros for one dose. The therapy is for children under two years of age who suffer from spinal muscular atrophy (SMA), which causes muscle wasting and, in severe cases, can lead to death if left untreated.

"There are no reasons to object to the hardship program," said the Paul Ehrlich Institute in Langen. The Federal Institute for Vaccines and Biomedical Medicines is responsible for approving such exemptions. This means that children who win the Novartis raffle can also be treated with the drug Zolgensma in Germany , although it is not yet approved in Europe.

Zolgensma not yet approved in Europe - raffle campaign is criticized

Affected parents, the Society for Muscular Illness and medical ethicists have criticized the raffle . According to medical criteria, the therapy should be available to those children who need it most, they say. According to the institute, one in 10,000 children in Germany is affected.

The drug * has been approved in the United States since May last year. In Europe, trials are still ongoing through the European Medicines Agency. Another drug is available for SMA patients , but it is less effective and does not help all children.

dpa

Read more : Only two million euros injection can save him: Most expensive therapy in the world for Baby John.

Seek help for rare diseases

To the photo gallery

* Merkur.de is part of the nationwide Ippen-Digital editors network .