Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels For Golodirsen Approval In DMD
While glodirsen did show higher levels of dystrophin production than eteplirsen did, increase is still unlikely to satisfy US FDA reviewers who disagreed with Janet Woodcock's decision to grant accelerated approval to eteplirsen.
You may also be interested in...
Sarepta cited two safety issues when announcing a complete response letter for its exon 53 skipping DMD drug golodirsen, but the US FDA's decision may be a result of the Exondys 51 approval from 2016, analysts suggest.
Pharmacy benefit manager’s new pipeline report offers insight into upcoming drugs of concern to payers.
A roundup of new applications submitted to FDA’s Center for Drug Evaluation and Research.