When Exondys 51 was approved to treat Duchenne muscular dystrophy, a deadly disease that puts kids in wheelchairs by the time they are teenagers, there was no proof the drug actually slowed the disease. That was seven years ago. The company still hasn’t provided conclusive data to this day.

The drug’s maker, Sarepta Therapeutics Inc., has so far reaped more than $2.5 billion in sales from Exondys 51 and two related drugs. All three were cleared via a US regulatory shortcut called accelerated approval that allows companies to market medications before completing definitive trials in cases where patients have few or no treatment options.

In the case of Exondys, Sarepta’s confirmatory study didn’t even begin until four years after it was approved and won’t be completed until 2024, eight years after it started selling the drug.

Complete your profile to continue reading and get FREE access to BenefitsPRO.com, part of your ALM digital membership.

Your access to unlimited BenefitsPRO.com content isn’t changing.
Once you are an ALM digital member, you’ll receive:

  • Critical BenefitsPRO.com information including cutting edge post-reform success strategies, access to educational webcasts and videos, resources from industry leaders, and informative Newsletters.
  • Exclusive discounts on ALM, BenefitsPRO magazine and BenefitsPRO.com events.
  • Access to other award-winning ALM websites including ThinkAdvisor.com and Law.com

Already have an account?


NOT FOR REPRINT

© 2023 ALM Global, LLC, All Rights Reserved. Request academic re-use from www.copyright.com. All other uses, submit a request to [email protected]. For more information visit Asset & Logo Licensing.

 

BenefitsPRO Broker Expo 2024Event

The premier educational and networking event for employee benefits brokers and agents.

Get More Information
 

BenefitsPRO Broker Expo 2024Event

The premier educational and networking event for employee benefits brokers and agents.

Get More Information
 

BenefitsPRO

Join BenefitsPRO

Don’t miss crucial news and insights you need to navigate the shifting employee benefits industry. Join BenefitsPRO.com now!

  • Unlimited access to BenefitsPRO.com - your roadmap to thriving in a disrupted environment
  • Access to other award-winning ALM websites including ThinkAdvisor.com and Law.com
  • Exclusive discounts on BenefitsPRO.com and ALM events.

Already have an account? Sign In Now
Join BenefitsPRO

Copyright © 2023 ALM Global, LLC. All Rights Reserved.