by Robin Fields
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Reporting Highlights
- A Record Price: The gene therapy Zolgensma helped children born with a fatal disease, spinal muscular atrophy, grow up to run and play. But the cost was stunning: $2 million per dose.
- Cashing In: While taxpayers and small charities funded the drug’s early development, executives, venture-capital backers and a pharma giant have reaped the profits.
- Priced Out: The drug’s cost adds to the nation’s ballooning bill for prescription drugs and puts Zolgensma out of reach for kids in many low- and middle-income countries.
These highlights were written by the reporters and editors who worked on this story.
Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma had ended and the forces that turned it into the world’s most expensive drug had taken over.
It was May 2014. He and his wife were sitting in the cafeteria at Nationwide Children’s Hospital in Columbus, Ohio.
