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The Eteplirsen Precedent: No Denying It – So Let’s Define It

 
• By Michael McCaughan

US FDA allowing Sarepta to seek accelerated approval for follow-on DMD agent – following 'precedent' set by eteplirsen. Now the new challenge is to define exactly what that precedent is.

It took almost 18 months to become clear, but it is now safe to say that the US FDA’s September 2016 approval of Sarepta Therapeutics Inc.’s eteplirsen for Duchenne Muscular Dystrophy is precedent-setting after all.

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More from Approval Standards

Moderna Pivots To Increase Focus On Cancer Amid US Vaccine Policy Changes

 
• By Joseph Haas

During its first quarter earnings call, Moderna avoided criticizing vaccine policy changes, but de-emphasized its flu/COVID-19 vaccine for those under age 50 and prioritized cancer programs.

Another Regeneron CRL Prompts Regulatory Operations Questions

 
• By Alaric DeArment

CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.

Postmarketing Surprise Is Latest Plot Twist For Novavax’s COVID-19 Vaccine BLA

 
• By Bridget Silverman

Three weeks after its user fee goal date, the FDA demanded a postmarketing commitment “to generate additional data,” Novavax said.

Gene Therapy Sponsor INTERACT, Pre-IND Meeting Confusion Lingers Despite FDA Guidance

 
• By Sue Sutter

PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.

More from Pathways & Standards

Another Regeneron CRL Prompts Regulatory Operations Questions

 
• By Alaric DeArment

CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.

New EU Filings

 
• By Neena Brizmohun

Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

US FDA Commissioner Rejected Some Proposed Reorg Plans

 
• By Elizabeth Orr

FDA Commissioner Martin Makary said in an interview that he will use the current structure to reform the agency. He also plans to address employee morale.