New gene therapy, to be priced at $4.25 million, has already transformed children’s lives Jason Mast Megan Molteni By Jason Mast and Megan Molteni March 20, 2024

https://www.statnews.com/2024/03/20/gene-therapy-orchard-therapeutics-metachromatic-leukodystrophy/?utm_campaign=morning_rounds&utm_medium=email&_hsmi=298983789&_hsenc=p2ANqtz-8tWUeLuJO_3vtVtsaHYv4eQ3KUv7wnEMRphDDaOzd4JL4ymEsGPhafvNbAp7FvA2KD7IVxsyFVYW8Czv-H_h8-Kzulcw&utm_content=298983789&utm_source=hs_email Victoria Rasberry sees the difference a gene therapy can make every morning. Her 8-year-old daughter Addi needs thick mucus sucked out of her airways so she can breathe. She eats breakfast from a feeding tube and must take 28 different medications to prevent pain, infections, and seizures. Her brother Oliver, age 3, is a typical giggling toddler. The children share a genetic mutation that causes a fatal neurodegenerative disease called metachromatic leukodystrophy, or MLD, but as a baby he was treated in Italy with what its maker said today will cost $4.25 million in the U.S., making it the most expensive drug in history. “When Addi was this age she had lost the ability to speak and was already using a ventilator to sleep at night,” Raspberry said. “It’s amazing to see how well Ollie is doing in comparison. And it’s all because of the gene therapy.” On Monday the U.S. FDA approved Orchard Therapeutics’ gene therapy Lenmeldy, an achievement decades in the making from parents’ efforts, researchers’ experiments, and a company’s experience with other gene therapies that failed. Now there’s the hurdle of that price. “It may take months sometimes to get those things worked out,” Paul Orchard of the University of Minnesota said about insurers. “And some of these kids aren't going to have months to screw around.” STAT’s Jason Mast and Megan Molteni have more on the complicated story. https://www.statnews.com/2024/03/18/metachromatic-leukodystrophy-nerve-disease-children-gene-therapy-orchard-lenmeldy/?utm_campaign=morning_rounds&utm_medium=email&_hsmi=298983789&_hsenc=p2ANqtz-_g-Yi4szim62kaksSiUY1SjB4RzZADinLA1bSfOgCEQqBFzAbOp3kHeGJg25GvfYOnIam6-J3QwLmrOeyLoaQxfXQS1Q&utm_content=298983789&utm_source=hs_email