When a $2.1 Million Drug Could Cure Your Child’s Fatal Disease

Gene therapy is bringing out the best in America’s health-care system—and its worst. Zolgensma, the first systemic gene therapy of its kind that the U.S. Food and Drug Administration has approved, appears to cure in one shot a rare muscle-destroying disease that can be a death sentence for infants and toddlers. (The kids still carry the gene mutation; they just don’t exhibit the eventually fatal symptoms.) It’s also the world’s most expensive medication. Novartis AG set the price at $2.1 million after the FDA approval came down on May 24, and some families have been left scrambling for ways to get the drug in its first several months on the market.

The medical system always faces a learning curve with a new treatment, particularly one this revolutionary, but the stakes for patients who could benefit from Zolgensma have made things that much tougher. Only 400 American babies a year are born with spinal muscular atrophy (SMA), but left untreated, it kills them before they turn 2 years old.