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.
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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.
PTC Therapeutics argues FDA did not apply the same standard to Translarna in Duchenne muscular dystrophy as previous DMD products, but FDA says accelerated approval was not possible because of negative efficacy data.
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.