It is one of the largest hereditary diseases, spinal muscular atrophy. Children born with the most severe type 1 form can neither sit on their heads nor return. They have difficulty breathing, swallowing and coughing. Almost all children die before their second birthday. Swiss pharmaceutical company with a new therapy Novartis Affected children and parents now hope to recover. Presents Basel at a London conference company A gene therapy called AVXS-101, which must be a great success. Novartis hopes to offer treatment in the EU, USA and Japan in mid-2019.
Novartis confirms the early editing dossier with the compelling results of the first clinical trial involving 15 children. Two years later, everyone was still alive. Eleven out of eleven children were able to sit, eat and talk normally. We can go back to nine – and they can both run.
Gene therapy improves muscle atrophy
The effect of the AVXS-101 comes at a price – the most expensive treatment ever: Novartis managers estimated the value of a one-time infusion of four to five million dollars – this is almost many Swiss francs. Therapy can be done on the basis of outpatient clinic. And a single infusion is sufficient to achieve the desired effect.
Spinal muscular atrophy is a muscle loss. Since the nerve cells in the spinal cord are damaged, the muscles receive very little impulses or disappear – they disappear. The cause of the disorder is a faulty protein. With the new treatment, doctors inject a healthy gene that can lead to the disease, the disease is treated causally. About 6,000 to 10,000 newborns are affected by severe form. In Switzerland, eight to twelve infants with this inherited disease are born each year.
To justify the surprising value, Novartis provided cost-benefit comparisons with other drugs. According to the estimates made by Novartis, AVXS-101 treatment leads to a good health condition for children over 13 years on average. Other drugs are more expensive with similar benefits than Novartis in a 10-year comparison.
"Four million dollars is a significant amount of money, but we believe that the cost-benefit ratio is maintained," says David Lennon, director of pharmaceutical company Avexis, before investors. Novartis recently acquired the company for nearly nine billion Swiss francs to secure new gene therapy rights.
There is no price limit for medicines in Switzerland.
When therapy is presented in Switzerland, it is clear. "At the moment we cannot comment on the plans for Switzerland," Novartis spokesman said. However, it can be assumed that the license will soon be implemented in Switzerland.
The new million therapy will fuel a controversy over the years. How much can a medication or therapy hold? What is the monetary value of human life?
In 2011, the Federal Supreme Court concluded that the upper limit was CHF 100,000 per year. This is in accordance with the results of the "End of Life" of the work of the National Fund: On average, a Swiss person is ready to pay 100,000 CHF a year for a patient with a cancer diagnosis.
These values are now turning to Novartis. But CEO Vasant Narasimhan promised: "We'll find a way around it medicine In Switzerland, there is no upper price limit for medicines and therapies. Maliyet It is theoretically possible to have a drug cost four million francs, Federal says the federal Public Health Department spokesman.
Created: 10.11.2018, 23:06 hours