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PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?

Executive Summary

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. 

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Not The End For Exondys In EU, Says Sarepta

Sarepta Therapeutics’ hopes of marketing its controversial Duchenne muscular dystrophy drug in the EU have again been dashed, but the company says it hopes to learn from EU regulators next year what it can do to get the drug to patients in Europe. Meanwhile, sales of the product are going well in the US.

Alkermes Refuse-To-File Reversal: Is It Really Good News For Anyone?

US FDA’s decision to 'un-refuse' ALKS 5461 was made within same review office that was caught up in the Sarepta controversy – albeit in a different division. What does the RTF reversal mean for Alkermes and for the agency?

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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