The most expensive drug in the world

Genetic therapy for Novartis spinal muscular atrophy costs more than $ 2 million a course.

The US Food and Drug Administration (FDA) last week approved the gene therapy for spinal muscular atrophy of Novartis Group (Switzerland). Gene therapy uses Zolgensma drugs , only need a single injection for $ 2,125 million. This is the " unprecedented" price for a drug.

In 2018, the initial price of Zolgensma offered by Novartis is up to 5 million USD per course. The Institute of Economic and Clinical Assessment (ICER), an independent organization in the US, said that the price was too high. On May 24, based on additional clinical data from Novartis as well as information and rates published by FDA, ICER believes that the price of Zolgensma is consistent with its effectiveness.

In response to complaints, Novartis' leader still defended Zolgensma's drug price, arguing it was more economical than long-term treatment that cost patients several hundred thousand dollars a year. The group is also working with insurance companies to reduce the burden on buyers.

Zolgensma is the most expensive drug in the world.(Photo: WINK News).

Spinal muscular atrophy (SMA) is a dangerous genetic disease with a rate of one in 10,000 births. Children with SMA are often paralyzed, have difficulty breathing, even die within months if they have SMA type I - the most serious form of the disease. An estimated 50-70% of SMA is type I.

Zolgensma uses a virus to produce a normal copy of the SMN1 gene, a defective gene in SMA. The drug is injected into the body only once.

FDA approved ZOLGENSMA based on clinical trials on 36 patients from two weeks to eight months of age. The agency said children treated with Zolgensma significantly improved motor development, including their ability to control their heads and sit up. According to Novartis leader, Zolgensma has so far treated 150 patients. Other tests are still underway.

Side effects of Zolgensma are high liver enzymes and vomiting. FDA now requires manufacturers to add to the drug label warning of serious liver damage.

Novartis CEO Vas Narasimhan commented that Zolgensma is almost cured of SMA if the child is injected immediately after birth. However, the data indicate the drug effect lasts about 5 years.

Zolgensma competes with Spinraza of Biogen Inc, the first FDA-approved SMA treatment. Patients using Spinraza must deliver the medicine to the spine every four months. The list price of this therapy is USD 750,000 for the first year and USD 375,000 for the next year. These prices are also deemed too high by ICER.

Novartis expects Europe and Japan to approve Zolgensma by the end of the year.