The most expensive drug in the world is already sold

Zolgensma works by providing a functional copy of the defective gene responsible for SMA, which stops the progression of the disease by a single intravenous infusion.

SMA is a degenerative disease of genetic origin that affects one in 10,000 people.

The US Food and Drug Administration said the safety of the drug had been tested in an ongoing and completed clinical trial involving 36 patients aged between two weeks to eight months. The strongest evidence of its effectiveness is based on the results of the current trial, which revealed that "patients treated with Zolgensma demonstrated a significant improvement in their ability to achieve developmental milestones", including a control head and arms. Sit without support.

Jerry Mendell, a physician involved in the trial conducted at Nationwide Children's Hospital in Columbus, Ohio, added that the level of efficacy, offered as a punctual treatment, "is truly remarkable and offers an unprecedented level of hope to families. "

The most common side effects of Zolgensma are an increase in liver enzymes and vomiting, the FDA said. Genetic and cellular therapies use biology to reverse diseases ranging from conbad blindness to childhood leukemia.

When there is no other choice

Some good results

Patients treated with the drug Zolgensma showed significant improvements in their ability to complete important stages of motor development, including head control and the ability to sit unaided.

We knew it was going to be expensive

The Swiss pharmaceutical company that designed the treatment had argued that the value of Zolgensma could be between $ 1.5 and $ 5 million, but had already set a definitive price of $ 2,125,000.