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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.

The circumstances surrounding that approval were obviously unprecedented from the first. The drug was approved because Center for Drug Evaluation...

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MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 
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More from Pathways & Standards

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Nipah Vaccine With ‘Pandemic Potential’ Wins EMA PRIME Designation

 
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The developers of the ChAdOx1 NipahB vaccine will be able to benefit from the early and enhanced support that the European Medicines Agency offers through its priority medicines scheme.

Elegy For SACHRP: US HHS Research Protection Panel Tackled Tough Pediatric Trial Questions

 
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The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.