Sarepta Therapeutics Off To A Strong Start With New Name And Data

The RNA drug-discovery company, formerly AVI BioPharma, is reinventing itself as an orphan disease drug specialist called Sarepta Therapeutics. With the release of positive Phase IIb data on its lead candidate for Duchenne muscular dystrophy, the transformation has gotten a big boost.

With a new name and positive Phase IIb data on its lead development compound – an RNA-based drug for the treatment of Duchenne muscular dystrophy – Sarepta Therapeutics Inc.is in the midst of transforming itself into an orphan disease specialist. The company, formerly called AVI BioPharma, was known for years mainly as an RNA technology and drug-discovery company, albeit one that struggled to sign lucrative partnerships with big pharma like some of the leading pioneers.

The company changed course to embrace an orphan disease business model, focused on its early clinical-stage DMD program, under the...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Archive

Ocaliva: Still No Clarity On Why EU Court Opposed Revocation Of Approval

 

Advanz Pharma would have had to show that the European Commission’s decision to revoke Ocaliva’s conditional marketing approval risked causing serious and irreparable harm, according to lawyers from Van Bael & Bellis.

Final Chance To Have Your Say: Take Our Reader Survey This Week

 
• By 

This is your final call to participate in the survey to better understand our subscribers’ content and delivery needs. The deadline is 20 September.

Shape Our Content: Take The Reader Survey

 
• By 

We are conducting a survey to better understand our subscribers’ content and delivery needs. If there are any changes you’d like to see in coverage topics, article format, or the method in which you access the Pink Sheet – or if you love it how it is – now is the time to have your voice heard.

Brazil Pilots Digital Drug Pack Inserts

 

A new pilot aims to take Brazil closer to ‘digital transformation.’

More from Pink Sheet

Expedited Reviews For China-Originated Drugs Shift Back Home

 
• By 

The proportion of China-originated drugs granted expedited reviews in China doubled in the first half of this year, Pharmaprojects data show, surpassing those receiving similar treatment in the US and likely driven by multiple factors.

Sarepta Will Resume US Elevidys Shipments For Ambulatory DMD Patients After FDA Reverses Course

 
• By 

The FDA recommended removal of a voluntary hold on shipments of the gene therapy to younger patients after finding that the death of an 8-year-old boy in Brazil was unrelated to Elevidys. Discussions on safety of DMD treatment for non-ambulatory patients is ongoing.

EU 15% Tariff Deal Still Leaves Questions For Pharma On Possible Grace Period, Generic Exemption

 

While the 15% rate is a substantial blow for pharmaceutical firms, the US-EU deal provides a framework for trade agreements with other countries that could see the industry avoiding higher tariffs elsewhere.