A new treatment is approved for sufferers of a rare and deadly infant muscle disorder, but it comes with a record price tag: more than $2.1 million for a one-time dose.
U.S. regulators on Friday gave Swiss drugmaker Novartis approval for gene therapy drug - Zolgensma - to treat childhood spinal muscular atrophy, a leading genetic cause of death in infants.
Novartis told Reuters health and pharmaceuticals correspondent Caroline Humer - the price is right.
SOUNDBITE (ENGLISH): REUTERS HEALTH AND PHARMACEUTICALS CORRESPONDENT CAROLINE HUMER, SAYING: "Novartis says that it is worth $2.125 million dollars because right now there are treatments on the market that costs almost $500,000 a year already.
In this first trial for children who have gotten it, the results have been pretty amazing.
Everybody agrees that children who had been predicted not to be able to walk, or even swallow, or even lift their heads can move around, some of them can run, they can eat on their own.
So compared to what the disease is, it's been somewhat miraculous they say." The record price tag is sure to raise eyebrows among politicians and consumer groups already upset over sky-high costs for prescription drugs.
SOUNDBITE (ENGLISH): REUTERS HEALTH AND PHARMACEUTICALS CORRESPONDENT CAROLINE HUMER, SAYING: "Already I think we're starting to hear a little bit on Twitter and we've gotten a bunch of emails from patient advocates saying that this is just too high of a price.
The company has said insurers, the payers, the big ones are ready to pay for this." But one independent group, The Institute for Clinical and Economic Review, after saying the price was too excessive, changed its mind Friday, seemingly convinced by data on the drug's live-saving results.
Novartis is not alone in treating the disease.
Biogen has a drug which requires multiple doses starting at $750,000 the first year and $375,000 each year thereafter.
And rival Roche is working on its own treatment, which it plans to seek approval for - later this year.
No word yet on how much that drug will cost.