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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US FDA sees parallels in ataluren’s development in Duchenne and firm's unsuccessful efforts to find efficacious subsets in cystic fibrosis.
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.
Keeping track of US FDA approvals last week included watching the creative and the familiar.