New Drug Could Cure Horrible Disease. Now, Brace Yourself

Novartis AG is expected to sell Zolgensma for $2M per treatment
By Neal Colgrass,  Newser Staff
Posted May 7, 2019 1:00 PM CDT
Updated May 11, 2019 4:53 PM CDT
New Drug Could Cure Horrible Disease. Now, Brace Yourself
In this 2016 photo, Swanville High School student Ethan Och demonstrates how he uses an iPad and an amplifier to continue to play percussion in band even with his physical limitations due to spinal muscular atrophy in Swanville, Minn.   (Dave Schwarz/St. Cloud Times via AP)

(Newser) – A new wonder drug is coming for babies with a crippling, often fatal disease. Expected price tag: $2 million per treatment, the Wall Street Journal reports. Novartis AG is expected to begin selling Zolgensma, a possible cure for spinal muscular atrophy, after the FDA approves it this month. Novartis executives say Zolgensma's ability to curb SMA—a muscle-wasting disease that often kills babies before age 2—explains the eye-popping price. But it is controversial: "A therapy is useless if no one can afford it," says a health-insurance industry spokeswoman. Insurance companies are also uneasy about paying for a purported cure when no one knows about the long-term effects. After all, Zolgenma's first clinical trial just happened five years ago.

Zolgensma is one of several new gene-therapy drugs that target a faulty gene behind a horrible illness. The only other US-approved gene therapy, Luxturna, costs $850,000 and helps children with a rare eye condition, per the Journal. Others include "bubble-boy disease" drug Strimvelis ($663,000) and leukemia drug Kymriah ($475,000). What's more, Bloomberg reports that Zolgensma seems to help a wider group of patients whose motor function is slowly ruined by SMA. But parents are bracing for a battle if the insurance industry balks at the price. "It'll be a big fight," says Louisiana resident Andrea James, whose infant son is already taking spinal injections for SMA. Zolgensma's actual price is yet to be announced, and the $2 million figure is just an estimate; it could go as high as $5 million. (Read more Big Pharma stories.)

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