Mechanism-Based Drug Rejection? Ataluren’s Unlikely Personalized Medicine Milestone
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.
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Cystic Fibrosis Failure Weakens PTC's Duchenne Argument, FDA Says
US FDA sees parallels in ataluren’s development in Duchenne and firm's unsuccessful efforts to find efficacious subsets in cystic fibrosis.
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.
Kalydeco Expands Indication Without Clinical Data; Keytruda Is Latest Bladder Cancer Approval
Keeping track of US FDA approvals last week included watching the creative and the familiar.