Novartis Creates Cure For Spinal Muscular Atrophy. Its Cost? $2M

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Within the upcoming days, pharmaceutical giant Novartis is expecting approval for what it calls the first “blockbuster” gene-replacement cure. According to the MIT Technology Review, a blockbuster drug is any that brings in more than $1 billion in annual sales.

But the gene therapy for infantile spinal muscular atrophy (SMA) type 1, Zolgensma, comes at a shocking $1.5 to $2 million dollar price tag, making it one of the most expensive one-time treatments ever.

Type 1 SMA is a degenerative disease that is typically fatal within two years.

The drug company will reveal the price as soon as it receives final US Food and Drug Administration approval. The company expects a favorable decision this week.

Gene therapy is a treatment method in which viruses insert healthy genes into the cells of a patient, and, while the concept has yet to see many large-scale uses, experts say that it could cause a revolution for the future of US health care.

According to FDA estimates, by 2025, roughly 10 to 20 cell or gene therapy treatments will be released each year. New treatment options could cause new problems for American health insurance companies, especially because of their price tags.

“The issue with many of these drugs is that they are expensive but also life-changing for those who need them,” said Harvard Pilgrim Health Care chief medical officer Michael Sherman. For this and other gene therapies coming down the pike, it’s not about saying no, but how do you say yes without bankrupting the system?”

Novartis executives say that included in the launch of their SMA drug will be a “pay-as-you-go” program in addition to reimbursement options if patients die or require additional assistance.

“We’d like to pay over time,” Sherman said, “and to stop paying if it stops working. At these ever-increasing prices, I think it’s fair to ask.”

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