PTC Therapeutics won’t be getting a decision soon from European regulators on its effort to get an extension of its conditional approval for Translarna (ataluren) as a treatment for Duchenne muscular dystrophy. The biotech announced this morning that the regulatory assessment won’t be completed mid-year, adding that execs expect “that Translarna’s current marketing authorization status will remain valid until a decision is adopted by the European Commission.”
PTC is also submitting a draft clinical protocol for regulators to look at, maintaining their confidence that they can get their marketing OK after they agree to conduct a new clinical trial.
PTC shares $PTCT dropped about 7% on the delay.
The CHMP had already raised a “major objection” to Translarna, zeroing in on whether or not the drug could be considered effective based on the failure of the Phase III study, according to PTC’s last quarterly report. PTC $PTCT managed to get a conditional approval to sell Translarna in Europe after the Phase IIb study failed as well. PTC won over the Europeans, though, through some post hoc analysis that PTC claimed demonstrated a benefit for some patients, as well as a clean safety profile.
This delay for PTC comes on the heels of a steady drumbeat of bad news for the Duchenne muscular dystrophy community. The EMA turned thumbs down on BioMarin’s application for drisapersen, after a rejection by the FDA, which prompted the company to scrap its effort and bury three other experimental drugs to boot.
The last high-profile DMD drug with a shot at a near-term approval is eteplirsen, which Sarepta has been pushing for an accelerated approval with data from only a dozen patients. FDA insiders have all but ridiculed the data, but in the face of an intense lobbying campaign by patient advocates and families, the agency has delayed its final decision twice. Now they’re waiting to see if a batch of new patient assessments can demonstrate some sign of dystrophin production, which may indicate that it could have a positive impact on the lethal disease.
The EMA has been tight-lipped about the process, but PTC’s last 10-Q reported that the CHMP had called a scientific advisory group together to take another look at the drug.
The UK’s drug watchdog NICE recently surprised analysts with a recommendation for Translarna. And they’ve reportedly been negotiating over the price, which may now be a moot consideration
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