The FDA and the EMA go their separate ways on DMD: CHMP slaps down Sarepta but offers rival PTC an expanded market

European and US regulators occasionally take completely conflicting stands on drugs, and nowhere is that more evident than in Duchenne muscular dystrophy.

Today, Europe’s CHMP turned thumbs down on Sarepta’s $SRPT Exondys51 (eteplirsen) two years after the FDA approved it for marketing in one of the most controversial decisions in the agency’s history. And the same group embraced an expansion of PTC’s $PTC ataluren, even though the drug has failed repeated clinical trials and has been snubbed now several times by the FDA for its unproven efficacy profile.

Neither Sarepta nor PTC have gotten this far this fast without a willingness to dig in deep and fight it out every step — and true to form Sarepta will now challenge the CHMP’s decision and look for a reversal.

That’s unlikely, but Sarepta has bucked the odds before.

PTC, meanwhile, can celebrate its advance with a thumbs up on expanding its drug to patients as young as 2 who suffer from nonsense mutation DMD.

Ironically, there are plenty of industry execs who believe that neither drug should be on the market with the data that’s been available so far. But they don’t get a vote.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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