NPR

At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever

The Food and Drug Administration approved a new gene therapy for a rare but devastating genetic disorder. The drugmaker says the cost is worth it because it's a one-time treatment that saves lives.
Zolgensma, a new drug approved by the FDA Friday, costs more than $2 million. Source: Novartis

The federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the most expensive drug on the market. It costs $2.125 million per patient.

But for those patients lucky enough to get it, it appears it can save their lives with a one-time treatment.

Three-year-old Donovan Weisgarber is one of those patients. When he was born he seemed perfectly healthy. But within weeks, it became clear something was terribly wrong.

"It was about when he was about one month old that when

You're reading a preview, sign up to read more.

More from NPR

NPR2 min read
Imelda Causing 'Major, Catastrophic Flooding' In Southeast Texas, Forecasters Warn
Some parts of southeast Texas could see rain totals of 25 to 35 inches through Friday, the National Weather Service says.
NPR7 min read
Jon Pardi's Hyperactive Honky-Tonk Brings The Past Into The Present
Heartache Medication favors muscled-up guitars and booming drums, but reserves a place of honor for gutsy, tunefully expressive fiddle and steel solos.
NPR2 min readPolitics
UAE Agrees To Join U.S.-Led Maritime Coalition To Protect Gulf Shipping
The contingent was organized by Washington following attacks in June on oil tankers in the Strait of Hormuz that it blames on Iran. Britain, Australia, Saudi Arabia and Bahrain have also joined.