Drug Development

PTC shares plunge anew after FDA slaps down Duchenne drug for a second time

PTC Therapeutics CEO Stuart Peltz

PTC Therapeutics CEO Stuart Peltz

There will be no Lazarus-like rise from the dead for PTC Therapeutics at the FDA — at least for now. The agency has slapped down the biotech’s first appeal of the FDA’s refusal to even consider what it has already concluded was an inadequate application for its Duchenne muscular dystrophy drug Translarna (ataluren).

The news drove PTC’s shares $PTCT down 40% by the end of the day, as investors bet against any approval in the near future.

The FDA’s controversial decision to approve Sarepta’s Exondys 51 (eteplirsen) for DMD has prompted BioMarin $BMRN to consider making an appeal on the FDA’s rejection of drisapersen, as Endpoints News reported on Thursday. But PTC hadn’t waited for that decision before starting its own campaign to get in front of regulators and experts to make its pitch.

Back in February the FDA had issued a refuse-to-file notice on ataluren, rejecting the application at the outset. But even with this second rejection, the biotech isn’t backing off. Instead, PTC says it is prepping for another appeal to “the next supervisory level of the FDA.”

Like Sarepta $SRPT, PTC wants a chance to get patient advocates in front of regulators to help make their case. And some are ready to go.

“This inconsistency is unacceptable and is concerning for the entire community,” said Pat Furlong, founder of Parent Project Muscular Dystrophy, in a statement. “This devastating, muscle-wasting disease cuts short the lives of boys and young men and every day that we wait for treatments, is a day in which muscle function is lost and not regained. As a community, we cannot rest until there are treatments for all the boys and young men.”

PTC has already received conditional approval in Europe, despite the fact that the drug has failed clinical studies for DMD. But the biotech has never stopped insisting that it has identified positive data that would support an approval.

Gena Wang at Jefferies says she’s not surprised, and given the recent activity among European regulators, she thinks PTC has plenty to worry about on that front as well.

“CHMP issued a request for supplemental information (RSI) including a major objection for ataluren review, suggesting high risk for PTCT to renew the EU marketing authorization,” she noted this morning.  “Following an oral explanation meeting regarding the renewal of ataluren EU marketing authorization (conditional approval in 2014), CHMP issued a RSI, including a major objection pertaining to ataluren’s efficacy, overall risk-benefit profile and design/conduct of an additional clinical trial to provide comprehensive clinical data. Additional concerns related to the primary pharmacology of ataluren and label matters were also raised. It would be PTCT’s responsibility to fully address concerns raised by CHMP and the company expects an opinion from CHMP by YE16. We continue to see high risk of renewing ataluren’s EU authorization.”

“We believe that fair consideration of the totality of Translarna’s data requires a full review of our application by the FDA,” said PTC CEO Stuart W. Peltz. “In light of this, continuing the formal dispute resolution process reflects our ongoing commitment to work with regulators and the Duchenne community to make Translarna available to nmDMD patients in the United States.”

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