Drug Development

PTC sees higher regulatory risk in fight to keep its Duchenne drug on the European market

PTC Therapeutics $PTCT hasn’t yet lost the right to market its failed Duchenne muscular dystrophy drug Translarna in Europe. But the biotech’s exec team is telling investors that they have waning hopes of continuing to make it available as regulators ponder whether they’ll renew the authorization.

In a statement out Wednesday the company noted:

Following the company’s CHMP meeting in October, the committee requested additional information regarding the risk-benefit profile of Translarna, its efficacy and the design of a potential trial that would provide comprehensive clinical data. While we have provided information in response to the CHMP’s requests and expect to continue to engage in further interactions, recent dialogue has introduced a higher degree of uncertainty as to the outcome. 

PTC obtained conditional approval to sell the drug in Europe more than two years ago, based on some retrospective analysis that claimed a significant benefit in walking distances for patients. The actual studies that PTC has completed so far, though, have failed. And the FDA refused to even consider its application in the US.

Here’s the take from RBC’s Simos Simeonidis:

Despite the sensitivities and difficulties that come with removing from the market an already available compound for a lethal pediatric disease for which there are no alternative therapies, we believe that the increasingly ominous language put forward by PTC, together with the fact that the confirmatory trial for Translarna clearly failed, make a recommendation for withdrawal from the market by CHMP a real possibility.

Janet Woodcock

Janet Woodcock

The snub from US regulators was appealed, though the agency quickly slapped down the first attempt. But the biotech isn’t giving up. PTC insists that it has good reason to have Translarna considered by an advisory committee, with plans to call on patient advocates to help make its case.

That route led to the controversial OK of Sarepta’s Exondys 51, though that arrived only after Janet Woodcock was allowed to overrule the agency review team as well as senior officials who vehemently objected to the approval, citing an absence of efficacy data and uncertainty over its safety.

PTC, meanwhile, says you can expect a final decision in Europe before the end of this year, which is just weeks away now.

Net product sales of Translarna hit $22 million in Q3, more than twice the amount it netted in the same period a year ago.

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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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