US regulators approve the most expensive drug of all time: $ 2.1 million to treat fatal diseases in infants


  • The US Food and Drug Administration has approved a $ 2.1 million medication to treat a fatal illness in children – the most expensive treatment of all time.
  • The drug, Zolgensma, from Novartis, is targeting a condition called spinal muscular atrophy that weakens the child's muscles so quickly that they are unable to move or breathe.
  • The disorder affects 400 babies born in the United States each year and kills most of them at 2 years of age.

US regulators have approved the most expensive drug of all time for a rare disorder that destroys a baby's muscular control and kills almost all those with the most common type of the disease within a few years.

The treatment costs $ 2,125 million. Direct costs for patients vary according to insurance coverage.

The drug, sold by Swiss drugmaker Novartis, is a gene therapy that treats a hereditary disease called spinal muscular atrophy. The treatment targets a defective gene that weakens a child's muscles so dramatically that they become unable to move and eventually unable to swallow or breathe. It reaches about 400 babies born in the US each year.

The Food and Drug Administration on Friday approved the treatment, called Zolgensma, for all children under the age of 2 years confirmed by a genetic test to have any of the four types of the disease. The therapy is a single infusion that takes about an hour.

Novartis said it would allow insurers to make payments over five years at $ 425,000 a year and give partial rebates if treatment does not work.

Independent review calls for price of $ 2.1 million "justifiable"

The other drug for the disease approved in the U.S.A is a medicine called Spinraza. Instead of a one-time treatment, it should be given every four months. Biogen, maker of Spinraza, charges a list price of $ 750,000 for the first year and after that $ 350,000 a year.

The independent non-profit group Institute for Clinical and Economic Review, which evaluates the value of expensive new drugs, has estimated that the price of new gene therapy is justifiable at a cost of $ 1.2 million to $ 2.1 million because "dramatically changes the lives of affected families." for this devastating disease. "

ICER chairman Dr. Steven D. Pearson called the treatment price "a positive outcome for patients and the entire health system."

The defective gene that causes spinal muscular atrophy prevents the body from producing enough protein to allow the nerves that control movement to function normally. Nerves die without protein.

In the most common type, which is also the most serious, at least 90% of patients die at 2 years of age, and those who are still alive need a ventilator to breathe. Children with less severe types become disabled more slowly and can live for up to a few decades.

Zolgensma works by providing a healthy copy of the defective gene, which allows nerve cells to start producing the necessary protein. This prevents the deterioration of nerve cells and allows the baby to develop more normally.

In patient testing, infants with the most severe form of the disease who received Zolgensma within 6 months of birth had limited muscle problems. Those who received the treatment earlier did their best.

Babies who took Zolgensma after six months have stopped losing muscle control, but the drug can not reverse the damage already done.

"She's walking and even jumping"

Evelyn Villarreal was one of the first treated children at eight weeks. His family, from Centerville, Va., Had lost their first child to spinal muscular atrophy at 15 months. Two years later, when Evelyn was born, a test showed that she also had the disease, so the family enrolled her in the gene therapy study at Nationwide Children's Hospital in Columbus, Ohio.

Evelyn is now 4 1/2 years old and has no muscle problems other than minor foot problems, said her mother, Elena Villarreal. She has been feeding for a long time, draws and talks well, and will begin kindergarten in the fall.

"She is very active and goes to the playground a lot," said Elena Villarreal. "She's walking and even jumping."

It's too early to know how long the treatment lasts, but doctors' hopes are increasing to last a lifetime, according to Nationwide Children's neurologist Dr. Jerry Mendell. Mendell led one of the first patient studies and is Evelyn's doctor.

"It's starting to look that way," he said, because some treated children who are now 4 or 5 years old are not yet symptomatic.

Early diagnosis is crucial, so Novartis has been working with states to make genetic testing for newborns needed at birth. The expectation is that most states will have this requirement until next year.

The FDA said the side effects include vomiting and possible damage to the liver, so patients should be monitored within the first few months after treatment.


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