The Eteplirsen Precedent: No Denying It – So Let’s Define It
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.
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US FDA Advisory Committee Reboot (Part I): Open Public Meeting A Place To Start?
CDER Director Patrizia Cavazzoni raised eyebrows when she stated an intent to remove the ‘emotional overtone’ of committee meetings. The open public hearing may be a place to start.
Aduhelm’s ‘Complex’ Circumstances Drove Extensive In-House Advice Process Before Accelerated Approval, Cavazzoni Says
Accelerated approval was considered as option in response to the negative advisory committee vote, but it was not a ‘default,’ CDER officials say. ‘It fit like a glove.’
PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?
US FDA appears willing to consider accelerated approval for PTC's Duchenne Muscular Dystrophy therapy ataluren if company is able to provide compelling evidence of increased levels of dystrophin in boys with nonsense-mutation DMD. That is quite a reversal from the agency’s view heading into PTC’s appeal.