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

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 we started to notice some symptoms," says his mother, Laura Weisgarber, 32, of Columbus, Ohio.

Donovan started getting really fussy, stopped squirming, and got weaker and weaker.

Donovan had spinal muscular atrophy (SMA), a rare disorder caused by a defective gene; the illness destroys the nerves that control muscles. Babies with the most severe form of the disorder typically don't live past their second birthday.

"We were devastated," Weisgarber says, recalling the diagnosis. "It was definitely the worst time of our lives."