New $2 Million Drug?

According to the Wall Street Journal, Zolgensma, a new gene therapy treatment for infant muscle-wasting disease, will soon be on sale for a potential cost of $2 million USD.

Executives of Basel-based Novartis have stated that Zolgensma’s potential to cure spinal muscular atrophy (SMA) justifies the expensive price tag. SMA affects between one to ten of every 10,000 live births, which adds up to approximately 500 new cases in the US, 600 new cases in Europe and 50 new cases in Japan each year.

Without treatment, babies suffering from the most severe form of atrophy typically die before their second birthday. Thus far, Zolgensma has shown promise. In clinical trials, babies treated with Zolgensma have lived beyond their second birthday and have hit key developmental milestones. It is expected that the US Food and Drug Administration will make a decision about Zolgensma’s approval by the end of May.

The head of Novartis’ Zolgensma program, David Lennon, has stated that media reports suggesting Zolgensma would be priced at $2 million USD are “complete speculation” and that the company is considering a price somewhere in the range of $1.5 million USD to $5 million USD. Any price point in this range would make Zolgensma one of the most expensive drug treatments.

Author: Daniel Seidman