At $2.1m a dose, this is the world’s costliest treatment for a disease

The US Food and Drug Administration on Friday approved a gene therapy for spinal muscular atrophy, a rare muscle-wasting disease that, in its most severe form, kills many babies before they turn 2.
The price will be $2.1 million, believed to be highest ever set for a one-time treatment. The therapy, to be sold as Zolgensma, alters the underlying genetic cause of spinal muscular atrophy and may permanently stop the disease. It is among the first of gene therapies that promise a cure for deadly inherited conditions.
“We feel we’re on a path where we hope one day to be able to bring SMA almost to elimination,” said Vas Narasimhan, the Novartis chief executive.
Many drugs for rare diseases have arrived on the market recently costing hundreds of thousands of dollars a year. But few have crossed $1 million.
Luxturna, a gene therapy that treats a rare, inherited form of blindness, costs $850,000, and Kymriah, Novartis’s onetime cell therapy treatment for leukemia, costs $475,000. The annual cost of some other rare-disease drugs can top $1 million, depending on a patient’s weight.
Novartis had declined until now to reveal Zolgensma’s price, but its executives have been on a monthslong publicity tour, making the case for a multimillion-dollar price tag.
At an event this week in Boston, for example, Narasimhan told reporters that Novartis planned to set a price that is “far lower” than the $4 million to $5 million the company had determined that the treatment is worth.
Critics say the statements were part of a sophisticated effort to acclimate the public to an astronomical price.
In an interview before the approval, David Lennon, the president of AveXis, the Novartis unit developing Zolgensma, said the company set the price based on a 50% discount off what Novartis believed it is worth, measured in part by the long-term cost of other rare-disease drugs.
Spinraza, a drug approved in 2016 that also treats spinal muscular atrophy, costs $750,000 in the first year of treatment and then about $375,000 a year after that. The cost of a decade of treatment with Spinraza, therefore, is more than $4 million.
Spinal muscular atrophy affects the motor nerve cells in the spinal cord, causing the body to lose physical strength and eventually robbing people of their ability to walk, talk, swallow and breathe.
About 1 in 11,000 babies are born with spinal muscular atrophy. Before Spinraza’s approval, there was no treatment.
FDA approved the treatment for children under 2 with all forms of spinal muscular atrophy.