U.S. Approval Of PTC Therapeutics' Translarna 'Remains Unlikely'

Shares of PTC Therapeutics, Inc. PTCT gained more than 13 percent Friday after the FDA found that the company's experimental drug to treat Duchenne muscular dystrophy may work.

The FDA's Advisory Committee meeting ruled that the body of evidence for the company's ataluren therapy was inconclusive by a 10 to 1 vote, Credit Suisse's Alethia Young said in a Friday note. This result was in-line with what the analyst was expecting, as it showed a positive benefit trend but wasn't sufficient enough to show any statistical significance.

"Based on the stock move with the briefing documents, we think that ataluren not getting approved in the U.S. is baked in," Young said. (See Young's track record here.) 

It's important to note that PTC's stock initially traded lower by more than 10 percent after the announcement on Thursday, but quickly recovered to erase the losses and then move higher Friday.

PTC will likely provide an update on protein level data at the World Muscle Society medical meeting on Oct. 5, according to Credit Suisse. Roche will likely begin pivotal studies on a therapy of its own later in 2017, according to the research firm. 

PTC's developments should have no read through to Sarepta Therapeutics Inc SRPT's Exondys 51's ongoing launch or potential development plans, Young said. Exondys 51 was "approved on the surrogate dystrophin endpoint that the FDA reiterated as valid."

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