Priority Review Politics: With Two Pending DMD Products, FDA Still Faces Community Anxiety
Duchenne muscular dystrophy advocates want updates on Sarepta's eteplirsen application; priority review for Marathon's deflazacort illuminates FDA's many considerations in agency’s ongoing effort to approve therapies for DMD.
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Steroid treatment like Emflaza is considered standard of care and availability of other products may make payer discussions difficult.
Emails reveal company wanted timelines and approval commitments from FDA toward the end of the eteplirsen review, saying it might not be able to continue studies; at the same time, Sarepta was telling investors it had 12 months of cash on hand.
Keeping Track: Janssen Gets A Breakthrough Therapy Status, FDA Awards Two Orphan Drug Designations, Portola Lands A CRL
The latest drug development news and highlights from our FDA Performance Tracker.