Zolgensma treats spinal muscular atrophy (SMA). This rare disease is associated with loss of mobility, muscle weakness and paralysis and is the leading genetic cause of infant death.
Although the US Food and Drug Administration (FDA) approved Zolgensma in 2019, some health insurance companies still do not cover it because of its high cost.
Worldwide, each year, more than 60,000 children are diagnosed with congenital spinal muscular atrophy. Zolgensma has been approved in more than 38 countries and about 1,000 children use it.
The pharmaceutical company Novartis confirmed that the drug has a high price because this is a one-time dose. Compared to the current therapy, Spinraza, which requires 10 years of treatment, the cost of Zolgensma is only half that.
Companies are racing to develop and market new drugs, but they are not taking the time to optimize the manufacturing process. As a result, drugs get to market quickly, but they are very expensive to manufacture and the cost is high when they reach the patient.
At a cost of $2.1 million per dose, Zolgensma - developed by AveXis, part of Novartis pharmaceutical company - is currently the most expensive drug in the US.