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

Trendingnow

1

Gen Z fled San Francisco for Texas and Florida. Now they’re turning ‘welcomer cities’ into the next big tech towns

2

Anthropic disables Fable and Mythos AI models after U.S. government bars it from giving foreigners access

3

Melinda French Gates' advice to new IPO millionaires: 'Give half your money away'

1

Gen Z fled San Francisco for Texas and Florida. Now they’re turning ‘welcomer cities’ into the next big tech towns

2

Anthropic disables Fable and Mythos AI models after U.S. government bars it from giving foreigners access

3

Melinda French Gates' advice to new IPO millionaires: 'Give half your money away'
Health

Drug Company Hits Pause on Launching an $89,000 Muscular Dystrophy Drug After Backlash

By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
By
Sy Mukherjee
Sy Mukherjee
Down Arrow Button Icon
February 13, 2017, 5:29 PM ET

Marathon Pharmaceuticals stared down some serious heat last week when it announced that it would sell a decades-old, cheap anti-inflammatory steroid for $89,000 to treat a heart wrenching rare muscle disorder in the U.S. The firm is having some second thoughts, pausing the drug’s market launch after massive public outrage and an investigation into the $88,000 price hike announced by Vermont Sen. Bernie Sanders and House Oversight Committee ranking Democrat Rep. Elijah Cummings on Monday.

The backstory: Illinois-based Marathon Pharmaceuticals won U.S. FDA approval of deflazacort, a common steroid that you can order for less than $1 per pill from abroad through online pharmacies, under the brand name Emflaza for a topline price of $89,000 for a year’s treatment. Why? The therapy wasn’t already approved in the U.S. as a steroid, and now it’s been cleared to help treat the symptoms of Duchenne muscular dystrophy (DMD) – a rare, muscle-wasting disease that leads to movement loss and eventual death in its young victims. Marathon had free reign over its pricing thanks to the lack of available Duchenne drug competitors.

The company took advantage of a number of U.S. programs meant to spur “orphan drugs,” or treatments for the kinds of disorders that afflict less than 200,000 Americans each year. For one, Emflaza’s approval to bolster muscle strength in DMD patients won the firm a coveted “priority review voucher” which it can sell for hundreds of millions of dollars to a pharma giant or use itself to shave four months off of the regulatory review period for another therapy. And Marathon barely did any actual clinical legwork to get the drug cleared for Duchenne – it relied on 1990-era clinical trial data before tacking on just enough study material to win an approval that doesn’t even address the root cause of the disease.

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

As I noted last week, this is an exact copy of Martin Shkreli’s modus operandi (Shkreli himself has now called out the similarities on his pharma critique/troll passion project Pharma Skeletons. “Bro. These guys invented price increases. I literally learned it from them,” he said of Marathon.)

The issue has inspired the wrath of patient advocates and lawmakers alike.

“Marathon did not develop deflazacort,” wrote Sanders and Cummings in a letter to Marathon CEO Jeffery Aronin, adding that they believe the firm is “abusing our nation’s ‘orphan drug’ program” and demanding answers for what they describe as an “outrageous plan” to gouge prices.

The firestorm appears to have hit home. Although Marathon has insisted that the $89,000 burden won’t be sloughed on to patients after discounts, rebates, and assistance programs, Aronin declared a temporary ceasefire on Monday.

“Since last week’s approval, we have heard both support from the community, and concerns about how the pricing and reimbursement details will affect individual patients and caregivers, such as how it effects coverage of other Duchenne products, such as EXONDYS 51,” wrote Aronin in a blog post for a Duchenne patient advocacy site. “Based on these questions, today we are announcing: We are pausing our launch which has not taken place yet… We have not sold any new product, and we will pause that process.”

The product that Aronin cites, Sarepta Therapeutics’ Exondys 51, is the only cleared treatment to treat the protein deficiency which causes Duchenne in the U.S., and has also enjoyed its fair share of controversy.

Just how long the “pause” will last is anyone’s guess. Pharma companies take pains to note that list prices are only topline numbers untethered from reality, and that they take pains to make sure vulnerable patients aren’t taking on those prices. For now, Marathon says that Duchenne patients and their families who buy deflazacort from overseas pharmacies at bargain prices may continue to do so.

About the Author
By Sy Mukherjee
See full bioRight Arrow Button Icon

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

U.S.’s screwworm fix is still a year away, risking more spread
North AmericaAgriculture
U.S.’s screwworm fix is still a year away, risking more spread
By Ilena Peng and BloombergJune 13, 2026
7 hours ago
Meet the lone star tick: the primary U.S. source of alpha-gal syndrome, which is a life-threatening meat allergy that’s on the rise
HealthHealth
Meet the lone star tick: the primary U.S. source of alpha-gal syndrome, which is a life-threatening meat allergy that’s on the rise
By Matthew Perrone and The Associated PressJune 13, 2026
16 hours ago
arms
HealthPsychology
You probably think you’re a really open-minded person, but the real thing raises your death anxiety
By Daryl Van Tongeren and The ConversationJune 12, 2026
1 day ago
AI was supposed to cut health care costs. One of its first jobs was charging you more, PwC report shows
AIHealth Care Service
AI was supposed to cut health care costs. One of its first jobs was charging you more, PwC report shows
By Whizy Kim and Tech BrewJune 12, 2026
2 days ago
Plunge All-in Cold Plunge Review (2026): Expert Tested
HealthDietary Supplements
Plunge All-in Cold Plunge Review (2026): Expert Tested
By Christina SnyderJune 12, 2026
2 days ago
Redwood Outdoors Duo Sauna Review (2026): Our Honest Experience
HealthDietary Supplements
Redwood Outdoors Duo Sauna Review (2026): Our Honest Experience
By Emily PharesJune 12, 2026
2 days ago

Most Popular

Gen Z fled San Francisco for Texas and Florida. Now they’re turning ‘welcomer cities’ into the next big tech towns
Real Estate
Gen Z fled San Francisco for Texas and Florida. Now they’re turning ‘welcomer cities’ into the next big tech towns
By Sydney LakeJune 13, 2026
21 hours ago
Anthropic disables Fable and Mythos AI models after U.S. government bars it from giving foreigners access
AI
Anthropic disables Fable and Mythos AI models after U.S. government bars it from giving foreigners access
By Jeremy KahnJune 13, 2026
1 day ago
Melinda French Gates' advice to new IPO millionaires: 'Give half your money away'
Startups & Venture
Melinda French Gates' advice to new IPO millionaires: 'Give half your money away'
By Emma HinchliffeJune 13, 2026
21 hours ago
SpaceX CFO Bret Johnsen quietly engineered its historic IPO and became an overnight billionaire
C-Suite
SpaceX CFO Bret Johnsen quietly engineered its historic IPO and became an overnight billionaire
By Sasha RogelbergJune 13, 2026
23 hours ago
U.S. energy secretary says 7 million barrels of oil exiting Persian Gulf daily, but Chevron CEO rebuts the claim
Energy
U.S. energy secretary says 7 million barrels of oil exiting Persian Gulf daily, but Chevron CEO rebuts the claim
By Jordan BlumJune 12, 2026
1 day ago
Trump expects to sign a deal with Iran on Sunday, but Tehran may want to avoid giving him a gift on his birthday
Middle East
Trump expects to sign a deal with Iran on Sunday, but Tehran may want to avoid giving him a gift on his birthday
By Jason MaJune 13, 2026
10 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.