by Linda A. Johnson
U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.
The treatment is priced at $ 2,125 million. Out-of-pocket costs for patients vary according to coverage.
The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy of inherited condition called spinal muscular atrophy. The treatment targets are so weak that they become unable to move, and eventually unable to swallow or breathe. It strikes about 400 babies born in the U.S. each year.
The Food and Drug Administration on Friday approved the treatment, called Zolgensma, for all children under age.
Novartis said it will let insurers make payments over five years, at $ 425,000 per year, and will give partial rebates if the treatment does not work.
The one other medicine for the disease approved in the US is a drug called Spinraza. Instead of a one-time treatment, it must be given every four months. Biogen, Spinraza's maker, charges $ 750,000 for the first year and then $ 350,000 per year after that.
The Independent Nonprofit Group Institute for Clinical and Economic Review, which rates the cost of new medicines, calculated that the price
ICER's president, dr. Steven D. Pearson, called the treatment's price. "
Spinal muscular atrophy normally. The nerves off the protein.
In the most common type, which is the most severe, at least 90% of patients by age 2, and any still need a ventilator to breathe.
Zolgensma works by supplying a healthy copy of the faulty gene, which allows nerve cells to start producing the needed protein.
In patients testing, babies with the most severe form of the disease who got Zolgensma within 6 months of birth had limited muscle problems.
Babies given Zolgensma after six months stopped losing muscle control, but the medicine can not reverse damage already done.
It is too early to know how long the benefit of the treatment Lasts, but doctors' hopes are rising. Jerry Mendell, a neurologist at Nationwide Children's in Columbus, Ohio.
"It's beginning to look that way," he said no symptoms.
Early diagnosis is crucial, so Novartis has to be tested with newborns at birth.
The FDA said that the patient has been treated for the first few months after treatment.
Spinal muscular atrophy drug may help children with later-onset disease
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FDA approves $ 2M medicine, most expensive ever (2019, May 24)
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