It is one of the worst hereditary diseases, muscular spinal cord atrophy. Children born with the heaviest type 1 type can neither sit nor hold their heads or turn. They have difficulty breathing, swallowing and coughing. Almost all children die before their second birthday. With a new treatment he gives the Swiss pharmaceutical company Novartis children and parents who are affected are now hoping for treatment. At a conference in London he presented Basel company a gene therapy called AVXS-101, which should achieve spectacular success. Novartis expects to be able to treat the EU, the US and Japan in mid-2019.
Novartis justifies early regulatory filing with exciting results from a first clinical study involving 15 children. After two years, everyone was still alive. Eleven of the twelve of the larger-dose children managed to sit, eat and speak normally. Nine could turn – and two could still run.
Gene therapy treats muscle atrophy
The impact of the AVXS-101 is priced – it's the most expensive treatment ever: Novartis executives appreciated the value of a one-time infusion at four to five million dollars – almost the same Swiss francs. Treatment can be performed in outpatients. And a single infusion is enough to achieve the desired effect.
Spinal muscular atrophy is a form of muscle wasting. Because the spinal cord nerve cells are damaged, the muscles receive little or no impulses – they disappear. The reason for the disorder is a defective protein. With the new treatment, doctors inject a healthy gene that can cure the disorder, the disease is being caused. Approximately one in 6,000 to 10,000 newborns is affected by the severe form. In Switzerland, eight to twelve babies with this hereditary disease are born every year.
To justify excessive value, Novartis offered a cost / benefit comparison with other medicines. According to Novartis, AVXS-101 treatment on average leads to 13 years of good health for children. Other drugs are more expensive, with similar benefits, according to Novartis in a ten-year comparison.
"Four million dollars is a significant amount of money, but we believe that the cost-benefit ratio is maintained," said David Lennon, head of the Avexis pharmaceutical company, ahead of investors. Novartis had recently bought the company for the equivalent of nearly nine billion Swiss francs – to secure the rights to the new gene therapy.
There is no price limit for medicines in Switzerland
When the treatment is offered in Switzerland, it is open. "At the moment we can not comment on the plans for Switzerland," says one Novartis spokesman. However, it may be assumed that the license will soon be sought in Switzerland.
The new treatment of millions will spark a debate that has erupted for years. How much does a drug or treatment cost? And what monetary value does a human life have?
In 2011, the federal Supreme Court concluded that the ceiling was CHF 100,000 per additional year of age. This is in line with the results of the End of Life National Fund study: on average, Swiss people are willing to pay CHF 100,000 for one year of cancer diagnosis.
These values are now turning to Novartis. However, CEO Vasant Narasimhan promised: "We will find a way around it medicine In Switzerland, there is no price cap for medicines and treatments. "It would be theoretically possible that a drug costs four million francs," says a spokesman for the Federal Office of Public Health.
Created: 10/11/2018, 23:06 watch