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By $ 2M, the priciest ever treat medicine lethal genetic disease

US. regulators have approved the most expensive medicine ever, for a rare disease that destroys the baby's muscle control and kills almost all those with the most common type of disease within a few years.

The treatment costs $ 2,125 million. Out-of-pocket costs for patients vary depending on insurance coverage.

The drug, sold by the Swiss pharmaceutical industry Novartis, is a gene therapy that deals with a hereditary condition called spinal muscular atrophy. The treatment aims at a defective gene that weakens a child's muscles so dramatically that they cannot move, and eventually cannot swallow or breathe. It beats about 400 children born in the United States each year.

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

Novartis said it will allow insurers to make payments over five years, to $ 425,000 a year and will give partial discounts if the treatment fails.

The other medicine for the disease approved in the US is a medicine called Spinraza. Instead of a one-time treatment, it must be given every four months. Biogen, Spinraza's manufacturer charges a list price of $ 750,000 for the first year and then $ 350,000 annually thereafter.

The independent nonprofit group Institute for Clinical and Economic Review, which values ​​the value of expensive new drugs, estimated that the price of the new gene therapy is eligible at a cost of $ 1

.2 million to $ 2.1 million because it " dramatically transforms families affected by this devastating disease ".

ICER president, Dr. Steven D. Pearson, called the treatment price "a positive result for patients and the entire health care system." which enables nerves that control the movement to function normally. Nerves die without protein.

In the most common type, which is also the most severe, at least 90% of patients must die at 2 years of age, and anyone living alive needs a breath ventilator. 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, allowing nerve cells to produce the necessary protein. It stops the deterioration of the nerve cells and causes the child to develop more normally.

In patient testing, children with the most serious disease form who received Zolgensma within 6 months of birth had limited muscle problems. The ones that were treated early were the best.

Babies who received Zolgensma after six months stopped losing muscle control, but the medicine cannot reverse the already damaged injury.

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

Evelyn is now 4½ years old and shows no muscle problems other than minor problems arose, her mother Elena Villarreal said. She has been feeding herself for a long time, she goes and talks well and starts the kindergarten in the autumn.

"She is very active and goes to the playground a lot," said Elena Villarreal. "She goes and jumps even."

It is too early to know how long the benefit of treatment lasts, but doctors hope that they can be lifelong, says Dr. Jerry Mendell, a neurologist at Nationwide Children's. Mendell led one of the early patient studies and is Evelyn's doctor.

"It is starting to look that way," he said, because a few children who are now treated 4 or 5 still have no symptoms.

Early diagnosis is crucial, so Novartis has been working with states to obtain genetic testing for newborns at birth. It expects most states to have that requirement next year.

The FDA said that side effects included vomiting and potential liver damage, so patients must be monitored the first few months after treatment.


Follow Linda A Johnson at https://twitter.com/LindaJ_onPharma .


The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education. AP is solely responsible for all content.

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