Patients Can't Clear Translarna's Data Hurdles As PTC Falls Short At FDA Panel
Patient testimony to the contrary, advisory committee members were not persuaded that PTC Therapeutics' ataluren, which failed its primary endpoint in two trials, is effective in treating nonsense mutation Duchenne muscular dystrophy.
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Keeping Track: A Burst of CRLs and BTDs, A Busy Week Of Submissions And Another Approval For Soliris
The latest drug development news and highlights from our US FDA Performance Tracker.
PTC Therapeutics appears to be hoping that CDER Director Woodcock will do for ataluren what she did for Sarepta's exon-skipping Duchenne muscular dystrophy treatment Exondys 51 since dispute resolution request would first go to Office of New Drugs, where Woodcock is acting director.
US FDA has shown it can be very flexible in expanding indications for a targeted therapy with a clearly established mechanism of action. But, as PTC Therapeutics learned, the agency’s willingness to apply mechanistic knowledge for targeted therapies cuts both ways.