It promises to be a largely normal life instead of a severe disability. The resources of the Novartis group, but it is so expensive that almost nobody can afford without the help of a health insurance Fund: $ 2.1 million.
Small, but mighty! – The drug Zolgensma helps fight against spinal muscular atrophy
The Swiss pharmaceutical group Novartis has received US FDA approval for a Gene therapy, with a cost of $ 2.1 million (1.87 million euros) per dose, the most expensive Drug in the world. The FDA approved the use of Zolgensma in children under two years of age for the treatment of the genetic disease spinal muscular atrophy (SMA). This leads to a progressive atrophy of the muscles and to an early death or life-long disability. The muscle disease occurs in about one of 10,000 live-born babies.
Once payment contra long-term costs
First of all, the high cost of goods in the authorisation procedures in the USA on the resistance of the authorities encountered. However, Novartis argued, apparently successfully, would need the application to save the life of patients who otherwise, on long-term treatments with a cost of several hundred thousand dollars per year.
The agent acts by a functioning copy of the defective gene provides. The course of the disease could be stopped with a one-time Infusion. Any existing damage can make the drug, however, reversed. Therefore, early diagnosis of SMA is important.
A different method of treatment: With an exoskeleton Affected are not necessarily reliant on a wheelchair
According to the neurologist Jerry Mendell, who led one of the first studies to patients, are the first children treated in the meantime, four to five years old and show no symptoms. Still, however, cannot say whether the one-off treatment to stop a lifetime.
Just another competition-medium
Previously it was used against the disease, only the means Spinraza of the U.S. biotechnology company Biogen. This Medicine needs to be administered every four months, fails in the first year of Treatment with 750,000 dollars to beech and, in consequence, with more than 375,000 dollars per year.
Novartis had provided for the now in the United States approved means least, a price range of 1.5 million to five million dollars in the room. Thus, the company was also a discussion about the costs of Gene therapies. An independent, not-for-profit institution, the rating of the value of more expensive new drugs, gave costs of between 1.2 and 2.1 Million U.S. dollars is justified. The Institute for Clinical and Economic Review argued, the resources to change the lives of affected families drastically.
Novartis provides health insurance according to their own information rates payments. The group expects this year with a permit in Europe and Japan. Experts estimate according to a survey that the group can generate with the funds until 2022 sales of two billion dollars. Biogen to trust you with Spinraza revenue of 2.2 billion dollars after it was 2018 1.7 billion. The Swiss Novartis ‘ rival Roche is working on a drug against the disease.
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