The most expensive medicine in the world has been launched on the market
A new remedy Zolgensma by Novartis Company is designed to replace long-term therapy for children with spinal muscular atrophy. The remedy is intended for a single use. Clinical trials that involved 36 children aged 2 weeks to 8 months have shown the effectiveness of such approach. The children demonstrated the acceleration of the development of motor skills (they better held their heads and sat). It should be reminded that spinal muscular atrophy often causes paralysis, breathing problems and death in the first months of a child’s life.
The uniqueness of Zolgensma lies not only in the effectiveness and possibility of abandoning chronic therapy. Currently, it is the most expensive medicine in the world - the cost of a single injection exceeds $ 2 million, SBS News reports. According to the developers, it is better to pay once than to spend the whole life of hundreds of thousands of dollars on medicines.
Zolgensma (onasemnogene abeparvovec-xioi) is a gene therapy for the pediatric patients under 2 years of the age with spinal muscular atrophy and mutations in SMN1 gene. The therapy does not deal with the symptoms, but with the genetic cause of the disease. The therapy involves the introduction of a working gene copy into the body to slow down the disease progression.