New Drug Could Cure Horrible Disease. Now, Brace Yourself

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. (More Big Pharma stories.)