• Home
  • Latest
  • Fortune 500
  • Finance
  • Tech
  • Leadership
  • Lifestyle
  • Rankings
  • Multimedia

Trendingnow

1

Now worth $200 million, Sarah Jessica Parker credits being ‘one of eight kids that struggled financially’ for her hunger, ambition, and work ethic

2

MacKenzie Scott alone accounted for one-third of America's $19.2 billion in megagifts last year

3

Amazon's record Prime Day masks a darker truth: Americans are spending more and getting less

1

Now worth $200 million, Sarah Jessica Parker credits being ‘one of eight kids that struggled financially’ for her hunger, ambition, and work ethic

2

MacKenzie Scott alone accounted for one-third of America's $19.2 billion in megagifts last year

3

Amazon's record Prime Day masks a darker truth: Americans are spending more and getting less
HealthBrainstorm Health

Brainstorm Health: Novartis $2.1 Million Drug, Brexit Fallout, Jakafi Drug Approval

By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
May 24, 2019, 5:34 PM ET
fortune logo icon (green)
Add Fortune on Google for similar content.

Happy Friday, readers!

On Friday, drug giant Novartis got the Food and Drug Administration (FDA) green light for a first-of-its-kind gene therapy to treat a rare, devastating genetic muscle-wasting disorder called spinal muscular atrophy (SMA). This victory was largely expected; all eyes, however, were on where Novartis would place the treatment’s list price.

Speculation was rampant. Would it be $2 million? $4 million? Even more? After all, Novartis CEO Vas Narasimhan had taken pains to argue that the therapy, Zolgensma, would be “cost-effective” at list prices nearing $5 million given SMA’s devastating nature and the fact that this treatment is a one-off medicine.

The company decided not to go quite that far in the end. But the number they’ve settled on still gives Zolgensma the distinction of being the world’s most expensive drug (at least by list price) at a $2.125 million tag.

Novartis has continued to assert this is a reasonable price, especially since it’s following it up with a number of unique payment programs. For instance, the company is offering an installment payment program to insurers who’d like to participate (and, technically, it’s “only” $425,000 per year over the course of five years, which is still cheaper than other available options, the firm’s executives say).

So what do the watchdogs have to say? Technically, the price tag falls within the higher range of cost-effectiveness pegged by groups like the Institute for Clinical and Economic Review (ICER), a persistent industry gadfly.

What will be more interesting to see going forward is what actual patients’ experience will be with the proposed pricing system, and what government regulators may do in the meantime. For now, welcome to the era of the world’s most expensive drug. And maybe don’t be shocked that the announcement comes ahead of a long weekend.

We’ll be off on Monday in observance of the Memorial Day holiday and back in your inboxes on Tuesday, May 28. In the meantime, read on for the day’s news and have a great Memorial Day.

Sy Mukherjee
@the_sy_guy
sayak.mukherjee@fortune.com

INDICATIONS

ICER revives Sarepta gene therapy debate. Speaking of drug pricing - ICER handed out another (and less positive) overview of a different pioneering medicine. The asset on the hot seat this time around is Sarepta's Exondys 51, which became the first approved treatment for the muscle-wasting disorder Duchenne muscular dystrophy a few years back (and to major controversy). ICER concludes that the treatment is not cost effective or, well, just generally all that effective for most Duchenne patients.

FDA approves Incyte drug for graft versus host. Biotech firm Incyte received its own important FDA approval on Friday, an expanded indication for Jakafi in treating a form of graft-versus-host disease. This is the first-ever medicine approved in this particular indication, and Incyte stock was up about 2% in Friday trading.

THE BIG PICTURE

Theresa May is out. What will Brexit mean for health care? With Theresa May announcing this morning that she'll resign as leader of the U.K.'s Conservative party and her role as Prime Minister, there are renewed questions about what Brexit will mean for the pharmaceutical supply chain. The answers are... Complicated. And they depend on a whole lot of issues that have yet to be settled (will there be an agreement set with the E.U., or a "no-deal" Brexit?) If it turns out to be the latter, public health officials have warned of potential disruptions to the pharmaceutical supply chain in the U.K. (which has already led some patients and drug makers to begin stockpiling medications).

REQUIRED READING

Theresa May Is Quitting Britain's Top Job. Business Won't Like What Comes Next, by David Meyer

What's Wrong With San Francisco, by Adam Lashinksy

The Best Business Documentaries to Watch This Weekend, by Polina Marinova

The Race Is On to Build a Better Battery, by Jeffrey Ball

Produced by Sy Mukherjee
@the_sy_guy
sayak.mukherjee@fortune.com
Find past coverage. Sign up for other Fortune newsletters.
About the Author
By Sy Mukherjee
See full bioRight Arrow Button Icon
Add Fortune on Google for similar content.

Latest in Health

Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025

Most Popular

Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Finance
Lorem ipsum dolor sit amet, consectetur adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam
By Fortune Editors
October 20, 2025
Fortune Secondary Logo
Rankings
  • 100 Best Companies
  • Fortune 500
  • Global 500
  • Fortune 500 Europe
  • Most Powerful Women
  • World's Most Admired Companies
  • See All Rankings
  • Lists Calendar
Sections
  • Finance
  • Fortune Crypto
  • Features
  • Leadership
  • Health
  • Commentary
  • Success
  • Retail
  • Mpw
  • Tech
  • Lifestyle
  • CEO Initiative
  • Asia
  • Politics
  • Conferences
  • Europe
  • Newsletters
  • Personal Finance
  • Environment
  • Magazine
  • Education
Customer Support
  • Frequently Asked Questions
  • Customer Service Portal
  • Privacy Policy
  • Terms Of Use
  • Single Issues For Purchase
  • International Print
Commercial Services
  • Advertising
  • Fortune Brand Studio
  • Fortune Analytics
  • Fortune Conferences
  • Business Development
  • Group Subscriptions
About Us
  • About Us
  • Press Center
  • Work At Fortune
  • Terms And Conditions
  • Site Map
  • About Us
  • Press Center
  • Work At Fortune
  • Terms And Conditions
  • Site Map
  • Facebook icon
  • Twitter icon
  • LinkedIn icon
  • Instagram icon
  • Pinterest icon

Latest in Health

Tru Niagen
HealthDietary Supplements
Tru Niagen Review (2026): Our Honest Thoughts
By Emily PharesJune 25, 2026
2 hours ago
ll
Economysummer
Deviled eggs, seltzer and a burger you can’t quit: The GLP-1 crowd is (halfway) reinventing the American BBQ
By Nick LichtenbergJune 25, 2026
6 hours ago
A pedestrian walks past a Gucci luxury fashion store at a shopping district on June 24, 2026, in Shenzhen, Guangdong Province, China.
RetailLuxury
Rich consumers taking GLP-1s are rebuying their wardrobes and eating smaller, fancier dishes—it’s a factor saving the luxury sector right now
By Eleanor PringleJune 25, 2026
9 hours ago
Ikea’s billionaire founder was so frugal that he bought clothes from flea markets and took free salt and pepper from restaurants
SuccessBillionaires
Ikea’s billionaire founder was so frugal that he bought clothes from flea markets and took free salt and pepper from restaurants
By Orianna Rosa RoyleJune 25, 2026
13 hours ago
How ‘Ozempic face’ is pushing Gen X, already the biggest Botox and filler consumers, to the facelift table a decade early
HealthGen X
How ‘Ozempic face’ is pushing Gen X, already the biggest Botox and filler consumers, to the facelift table a decade early
By Mia OsmonbekovJune 24, 2026
23 hours ago
The 4 Best Zinc Supplements of 2026: Expert Tested
HealthDietary Supplements
The 4 Best Zinc Supplements of 2026: Expert Tested
By Emily PharesJune 24, 2026
24 hours ago

Most Popular

Now worth $200 million, Sarah Jessica Parker credits being ‘one of eight kids that struggled financially’ for her hunger, ambition, and work ethic
Success
Now worth $200 million, Sarah Jessica Parker credits being ‘one of eight kids that struggled financially’ for her hunger, ambition, and work ethic
By Orianna Rosa RoyleJune 24, 2026
2 days ago
MacKenzie Scott alone accounted for one-third of America's $19.2 billion in megagifts last year
Success
MacKenzie Scott alone accounted for one-third of America's $19.2 billion in megagifts last year
By Sydney LakeJune 25, 2026
13 hours ago
Amazon's record Prime Day masks a darker truth: Americans are spending more and getting less
Retail
Amazon's record Prime Day masks a darker truth: Americans are spending more and getting less
By Nick LichtenbergJune 24, 2026
1 day ago
Ray Dalio just finished a 10-day trip to China. He says global leaders know America ‘doesn’t have what it takes to fight to maintain its empire’
Asia
Ray Dalio just finished a 10-day trip to China. He says global leaders know America ‘doesn’t have what it takes to fight to maintain its empire’
By Nick LichtenbergJune 24, 2026
1 day ago
After forcing workers back to the office, Goldman Sachs and JPMorgan Chase are now letting their staff work remotely—but only for the World Cup
Success
After forcing workers back to the office, Goldman Sachs and JPMorgan Chase are now letting their staff work remotely—but only for the World Cup
By Orianna Rosa RoyleJune 23, 2026
2 days ago
Ikea’s billionaire founder was so frugal that he bought clothes from flea markets and took free salt and pepper from restaurants
Success
Ikea’s billionaire founder was so frugal that he bought clothes from flea markets and took free salt and pepper from restaurants
By Orianna Rosa RoyleJune 25, 2026
13 hours ago

© 2026 Fortune Media IP Limited. All Rights Reserved. Use of this site constitutes acceptance of our Terms of Use and Privacy Policy | CA Notice at Collection and Privacy Notice | Do Not Sell/Share My Personal Information
FORTUNE is a trademark of Fortune Media IP Limited, registered in the U.S. and other countries. FORTUNE may receive compensation for some links to products and services on this website. Offers may be subject to change without notice.