The most expensive medicine in the world is intended for children
This money for the child suffering from spinal muscular atrophy was first raised through donations. However, the insurance company OZP then agreed to pay for the treatment of Max and two other children.
The drug was administered to the child at the Prague hospital "Motol". "This is the most expensive one-time drug we have ever worked with," said the hospital's representative Pavlina Dankova. How effective its action will be will become clear within the next three months.
Spinal muscular atrophy is a rare genetic disease caused by a mutation in the SMN1 gene (survival motor neuron 1). The lack of the protein encoded by this gene leads to dysfunction and death of motor neurons in the brainstem and spinal cord. As a result, muscle atrophy progresses and death occurs from respiratory failure.
Licensed in the USA and the European Union, Zolgensma is a gene therapy drug that is administered to the patient (children under two years old) intravenously only once. It is a normal copy of the SMN1 gene inserted into the genome of a special virus (because delivering genetic material into a cell is a natural ability of viruses). After administration, the defective gene is replaced with a normal one, and a sustained improvement in motor functions occurs.
As Vinegret.cz notes, the high cost of the drug is explained by the fact that the Swiss company Novartis released it not long ago, and it is aimed at a small group of patients – only a few thousand patients worldwide suffer from this disease. Therefore, all costs for the development and testing of the drug fell on their shoulders.