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

Trendingnow

1

26 Meta employees accuse Mark Zuckerberg of using AI to target 8,000 layoffs against workers on medical, parental or family leave

2

MacKenzie Scott, Melinda French Gates, and Lauren Sánchez Bezos are rewriting the rules of billionaire giving—one quietly, one strategically, one very publicly

3

After donating $48 billion to the Gates Foundation, Warren Buffett is quietly ending one of the biggest philanthropic relationships in history

1

26 Meta employees accuse Mark Zuckerberg of using AI to target 8,000 layoffs against workers on medical, parental or family leave

2

MacKenzie Scott, Melinda French Gates, and Lauren Sánchez Bezos are rewriting the rules of billionaire giving—one quietly, one strategically, one very publicly

3

After donating $48 billion to the Gates Foundation, Warren Buffett is quietly ending one of the biggest philanthropic relationships in history
HealthBiotech

This Biotech Just Gained Billions in Market Value Thanks to Its Cystic Fibrosis Drug Cocktail

By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
July 19, 2017, 4:15 PM ET
Cystic fibrosis drug campaign
Campaigners stage a protest outside Downing Street in central London, as part of a national campaign over the lack of availability of the "life-saving" cystic fibrosis drug Orkambi, which they want made available on the NHS. (Photo by Dominic Lipinski/PA Images via Getty Images)Dominic Lipinski - PA Images PA Images via Getty Images
Add Fortune on Google for similar content.

Vertex Pharmaceuticals (VRTX) is on a roll.

The Boston-based drug maker’s stock soared a stunning 21% in Wednesday trading after its triple drug combination therapy for cystic fibrosis (CF)—a rare genetic disorder that eats away at the lungs, pancreas, and other organs—posted promising results in early clinical trials.

Click here to subscribe to Brainstorm Health Daily, our brand new newsletter about health innovations.

Wall Street investors were hoping that Vertex’s cocktail could improve certain metrics of lung function by at least 2.5 percentage points, according to CNBC. The company says that it did that and more—to the tune of a 9.6 percentage point (or more) improvement.

That’s critical for a company with a rare disease focus. There are about 30,000 cystic fibrosis patients in any given year, but few actually have treatment choices. A regulatory win for Vertex’s combination therapy could prove critical.

The company’s financial success is also inextricably linked with pricing, as is the case with many “orphan drug” companies that make treatments for rare conditions. Vertex has had to wrangle with regulators in other countries over whether or not its CF drugs are worth the price. But promising results in such a hard-to-reach treatment space may be enough to guarantee regulatory success, especially if future trial results prove promising.

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

pete
PoliticsMilitary
Pete Hegseth wants to test troops for ‘testosterone deficiency’ — literally
By Konstantin Toropin, Matthew Perrone and The Associated PressJuly 15, 2026
9 hours ago
NordicTrack X24 Bike Review (2026): Expert Tested
HealthDietary Supplements
NordicTrack X24 Bike Review (2026): Expert Tested
By Christina SnyderJuly 15, 2026
14 hours ago
taco
HealthRestaurants
Taco Bell ‘voluntarily and temporarily’ cuts ingredients as investigators probe dodgy lettuce behind worst outbreak of its kind
By Mike Stobbe and The Associated PressJuly 15, 2026
14 hours ago
Billionaire Richard Branson says copying his 5 a.m. morning routine won’t make you successful—and will just cause ‘more burnouts than breakthroughs’
Successlifestyle
Billionaire Richard Branson says copying his 5 a.m. morning routine won’t make you successful—and will just cause ‘more burnouts than breakthroughs’
By Orianna Rosa RoyleJuly 15, 2026
21 hours ago
New experimental Alzheimer’s drug shows potential after study shows slower cognitive decline
HealthAlzheimer's
New experimental Alzheimer’s drug shows potential after study shows slower cognitive decline
By Lauran NeergaardJuly 14, 2026
1 day ago
A leadership consultant’s warning to managers: Don’t mistake grief for underperformance
Workplace CultureHuman resources
A leadership consultant’s warning to managers: Don’t mistake grief for underperformance
By Mikaela Cohen and HR BrewJuly 14, 2026
1 day ago

Most Popular

26 Meta employees accuse Mark Zuckerberg of using AI to target 8,000 layoffs against workers on medical, parental or family leave
Law
26 Meta employees accuse Mark Zuckerberg of using AI to target 8,000 layoffs against workers on medical, parental or family leave
By Barbara Ortutay, Alexandra Olson and The Associated PressJuly 15, 2026
15 hours ago
MacKenzie Scott, Melinda French Gates, and Lauren Sánchez Bezos are rewriting the rules of billionaire giving—one quietly, one strategically, one very publicly
Newsletters
MacKenzie Scott, Melinda French Gates, and Lauren Sánchez Bezos are rewriting the rules of billionaire giving—one quietly, one strategically, one very publicly
By Sydney LakeJuly 14, 2026
1 day ago
After donating $48 billion to the Gates Foundation, Warren Buffett is quietly ending one of the biggest philanthropic relationships in history
North America
After donating $48 billion to the Gates Foundation, Warren Buffett is quietly ending one of the biggest philanthropic relationships in history
By Marco Quiroz-GutierrezJuly 14, 2026
1 day ago
Jamie Dimon understands why people are anti-rich: 'We have, in fact, left the lower-income folks behind' and 'that's kind of annoying'
Economy
Jamie Dimon understands why people are anti-rich: 'We have, in fact, left the lower-income folks behind' and 'that's kind of annoying'
By Eleanor PringleJuly 15, 2026
17 hours ago
He sold his last company to Palantir. Now he's betting $32 million that robots can fix construction's labor crisis
Innovation
He sold his last company to Palantir. Now he's betting $32 million that robots can fix construction's labor crisis
By Lily Mae LazarusJuly 15, 2026
16 hours ago
FedEx CEO says we are in the middle of the biggest supply chain shift he’s seen in 35 years: ‘We are the referendum’
C-Suite
FedEx CEO says we are in the middle of the biggest supply chain shift he’s seen in 35 years: ‘We are the referendum’
By Fortune EditorsJuly 15, 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.