Did the FDA just leave the door cracked open for PTC’s Duchenne drug after repeated slapdowns?

At first glance, you might conclude that the FDA had simply done what it always does with PTC’s unending battle to gain a green light in the US for ataluren: slapped them down.

Stuart Peltz

This time, though, the biotech $PTCT says that regulators are pointing them down a potential accelerated pathway that could lead to an approval. And that would mark a step forward for a company that has recorded nothing but failure for this drug in the clinic over the years.

This possible path forward involves:

(A) re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients’ muscles, as quantified by procedures to be agreed upon between PTC and the FDA and using newer technologies.

That’s not a lot to go on, and the FDA of course is silent as a grave about whatever it may have told PTC. But the biotech says that as a result of their latest interaction with the FDA — in which they forced regulators to once again consider ataluren for an OK, then protested the repeat move to lock the door — they’ll be following up with the FDA on this new pathway and keeping US patients in an expanded access program to collect more data.

PTC CEO Stuart Peltz was able to win an accelerated approval in Europe and stay on the market even after repeated failures in the clinic for Duchenne muscular dystrophy. The FDA, meanwhile, was willing to give Sarepta an approval even though the company has yet to produce convincing efficacy data.

The big question now is whether the FDA — which has been signaling its willingness to open shorter pathways to an approval — left the door open just enough for PTC to eventually follow Sarepta into the market.

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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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