At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever

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Support for NPR and the following message come from Panera bread. Ed Pinera breakfast to go. No longer means to settle. Try their new maple glazed bacon. Scrambled, egg and cheese breakfast rap, and new Madda, gas carbon Ila cold brew Panera, food as it should be the food and Drug administration approved a new form of therapy today for a devastating genetic disease. It is providing hope for babies born with this rare but often fatal disorder. But as NPR health correspondent, rob Steiner reports at more than two million dollars. The treatment is the most expensive drug ever approved. When Donovan Weisgarber was born, he seemed perfectly healthy, but within weeks, his mom, Laura says it became clear something was wrong terribly. Wrong, it was when he is Abou one month old was, when we started to notice some symptoms, he started getting really fussy stop squirming, and got weaker and weaker turns out. Donovan had a genetic disorder spinal muscular atrophy. It was destroying the nerves that control his muscles many babies don't live beyond their second birthday. It's the most common genetic cause of death among infants. We were devastated obvious. Yeah. You know, obviously, you're devastated. Let's definitely the worst time of our lives. But then doctors told Laura and her husband Matthew about something new. They might be able to replace the defective. Gene killing Donovan with a new type of gene therapy, so they agreed to let doctors infused on of, and with genetically modified viruses, carrying healthy genes into his body. Donovan slowly started to improve and three years later. Donovan still needs a wheelchair in a feeding tube. But otherwise, is doing great. His mom says, and he loves going outside his family. He goes to preschool, so, yeah, he gets to do a lot of normal things. So it's just I mean it's amazing. Using and Donovan isn't alone. This gene therapy has been saving other babies with spinal muscular atrophy. David linen is the president of a vexes, the company that makes what's now called soul gems Ma first of all one hundred percent of the kids survived, and this is a population where ninety two percent of the kids would expect to have died or beyond permanent. Ventilation by the time they're twenty months old. But then we also saw additional things a lot of these kids could swallow seventy five percent of these kids were able to sit. And we actually had a few kids we're able to stand and walk independently based on these results Lennon's says the company is justified in setting the price at two point one two five million dollars for each child, which would make it the most expensive drug ever approved and that price tag is making a lot of jaws drop. You know it's absolutely stunning. Peter Bach studies health policy at Memorial Sloan cancer center in New York. It's just alarming that we have gotten to a point where, you know, any tree. That is a product of this collective scientific enterprise, that has grown out of the human genome project. That was publicly funded has now been captured by a single drug company and is now going to turn around and charge, potentially millions says, it's just the most extreme example of how drug prices are draining resources from society. We have been slowly, subjected to price increases the same way, the frog and the boiling water. Is slowly boil to death now. Linen acknowledges the price might team shocking, but he argues it's worth it. The only existing treatment for spinal muscular, atrophy clus, hundreds of thousands of dollars a year. And this will hopefully be a one time lifesaving treatment that will last a lifetime. What we're talking about it, and we have to remember as we're talking about a lifetime of benefit being condensed down into a one time treatment, we're not used to thinking about this that way, we're used to a system of chronic medication where we spread costs out over years, if not decades drug companies need to be able to recoup the cost of developing life-saving, cutting edge treatments. Limit says if they're going to be encouraged to find new breakthroughs Donovan's, parents didn't have to pay because their son was part of a research study. But they think the treatment is worth the price giving someone alive someone that would have died in infancy early childhood the opportunity to live into adulthood. I mean I don't know. I think that's valuable I think it's. Valuable investment rob Stein. NPR news.

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