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

 
• By Jessica Merrill

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...

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

 
• By Neena Brizmohun

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 Nielsen Hobbs

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 Nielsen Hobbs

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

 
• By Francesca Bruce

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

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US FDA Staff Stability Should Be A Congressional Priority, Former Chief Of Staff Says

 
• By Derrick Gingery

Lawmakers should address FDA staff upheaval and help the agency “get back on track,” former agency Chief of Staff Elizabeth Jungman said, while CBER Director Vinay Prasad told staff he is asking ex-colleagues to return to the agency.

US FDA ‘Commissioner’s Voucher’ As Public Relations Tool

 
• By Michael McCaughan

NRx Pharma is trying to capitalize on the headlines about a new US FDA hyper-fast review program, illustrating that the lack of details in the FDA’s announcement is an opening for sponsors to try to grab some attention.

HHS Wants Employees (And Public?) To Report Diversity, Equity And Inclusion Discrimination

 
• By Sue Sutter

A survey asks FDA staff to disclose whether DEI policies caused discrimination or resulted in disciplinary action against career employees over the past five years, but uses a web form that the general public also can access.