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Zolgensma from Novartis is the most expensive drug ever approved: Shots




Zolgensma, a new drug approved by the FDA on Friday, costs more than $ 2 million.

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Novartis

Zolgensma, a new drug approved by the FDA on Friday, costs more than $ 2 million.

Novartis

The Federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the most expensive drug on the market. It costs $ 2,125 million per patient.

But for those patients who are fortunate enough to get it, it seems that it can save their lives with a one-time treatment.

Three year old Donovan Weisgarber is one of the patients. When he was born he seemed quite healthy. But during weeks it became clear that something was very wrong.

"It was about when he was about a month old when we started to notice some symptoms," says his mother, Laura Weisgarber, 32, Columbus, Ohio.

Donovan began to become very fussy, stopped getting rid of, and became weaker and weaker.

Donovan had spinal muscular atrophy (SMA), a rare disorder caused by a defective gene; The disease destroys the nerves that control the muscles. Babies with the most severe form of the disorder do not usually live past their second birthday.

"We were destroyed," says Weisgarber, and remember the diagnosis. "It was definitely the worst time in our lives."

But then the doctors of Laura and her husband, Matthew, said about an experimental gene therapy that was tested for SMA. So they agreed to let them infect Donovan with genetically modified viruses that carry healthy copies of the gene he needed.

Donovan slowly began to improve. Donovan still needs a wheelchair to get around and a nutritional tube. But otherwise it goes well, says her mother.

"He loves to go outside. He loves to play with his family." He goes to preschool, says Weisgarber. "He'll do many common things. It's amazing."

And Donovan is not alone. Gene therapy called Zolgensma – has spared other babies with spinal muscular atrophy.

"We only saw remarkable results for these children," said David Lennon, president of AveXis, Inc., of Bannockburn, Ill., The company that developed Zolgensma. The company is owned by drug giant Novartis. (AveXis linked NPR with the Weisgarber family.)

Based on the AveXis studies, the FDA approved Zolgensma Friday, which only makes it the second gene therapy approved for a genetic disorder. Currently, about 700 patients are eligible for treatment, according to Novartis, and about 30 children are born each month with the disease.

Novartis set the price of $ 2,125 million, but offers insurance companies the opportunity to pay $ 425,000 a year for five years. This price tag makes Zolgensma the most expensive fabric approved.

"It's absolutely amazing," says Peter Bach, who studies health policy at the Memorial Sloan Kettering Cancer Center in New York.

Zolgensma's price tag, he says, is just the most extreme example of how drug prices drain community resources. The first gene therapy for a hereditary disease was approved in 2017 for a genetic form of blindness. It is also very expensive – $ 425,000 for each eye.

"We have been slowly exposed to price increases just as the frog in boiling water is slowly boiling to death," Bach says.

The insurance companies are expected to cover the costs. The company says that payment plans will be available.

AveXi's President Lennon acknowledges the figures may seem shocking. But he claims that the drug is easily worth it. The only existing treatment for spinal muscle atrophy, a substance called Spinraza, costs hundreds of thousands of dollars a year. Zolgensma will hopefully be a one-time life-saving treatment.

"We're talking about a lifetime benefit that is being condensed into a one-time treatment," says Lennon. "We are not used to thinking this way. We are used to a system of chronic medication where we spread things over the years not decades."

The drug is delivered as a single single dose to address the genetic cause of the disorder, the company said, producing long-term effects.

Lennon therefore calls it "very cost effective" and a "fair and reasonable price".

Drug companies must be able to recover the costs of developing life-saving, groundbreaking treatments, he says, if they are to be encouraged to find new breakthroughs.

"We continue to reinvest in new therapies beyond Zolgensma in the next wave of gene therapies that will hopefully cure other diseases," says Lennon.

At one point, Novartis publicly said that the price of Zolgensma could be as high as $ 5 million "The insurance companies should cover Zolgensma regardless of the cost," said Dr. Steven D. Pearson, president of the Nonprofit Institute for Clinical and Economic Review in Boston, an independent research group studying drug pricing. "That is a positive result for patients and the whole the health system that Novartis instead chose to price Zolgensma at a level that more equitably targets the benefits for these children and their families. "

ICER estimated that a reasonable price would be between $ 1.2 and $ 2.1 million per treatment, based on an estimate of years of quality life valued at $ 100,000 to $ 15,000 per year.

Donovan's parents did not have to pay for the treatment because their s eyes were part of a study. But they think Zolgensma is worth the price.

"I understand it can be shocking when you see a price tag on a treatment like this," says Weisbarger. "But to give someone a life – someone who would have died in childhood or early childhood the opportunity to live in adulthood – I think it's a valuable investment."

The company says it has produced the drug since January and deliveries of the drug will be released "shortly", says Lennon.



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