EMA, Regulatory

European regulators are locking the door on Sarepta’s Duchenne drug — but they aren’t throwing away the key

Sarepta Therapeutics $SRPT won’t be selling its Duchenne muscular dystrophy drug eteplirsen in Europe anytime soon.

European regulators at the CHMP confirmed their thumbs-down on the drug, Sarepta CEO Doug Ingram said, resisting the biotech’s attempt to change their position and setting up an all-but-certain formal rejection by the EMA.

Doug Ingram

Eteplirsen — sold in the US as Exondys51 — remains a highly controversial drug. Approved almost solely on the insistence of Janet Woodcock, who overrode objections from the review team and senior FDA officials, the drug’s label includes the note that the company has yet to prove its efficacy.

Ironically, the EMA has allowed PTC $PTCT to keep its rival Duchenne drug on the market in Europe, despite a woeful record of repeated trial failures that spurred the FDA to keep it barred from US patients.

But neither regulatory group has locked the door and thrown away the key.

“We were…encouraged by the openness of discussion with the SAG and CHMP and their willingness to engage on different approaches to provide additional data to support an eventual approval in Europe,” Ingram said in a statement. “Based on those discussions, Sarepta will work to explore a potential path forward that balances the needs of patients and their families to avoid lengthy and unnecessarily burdensome trials with those of European Regulators for additional supportive data consistent with existing European regulations. We will be seeking follow up Scientific Advice in 2019 in order to explore the approach to bring eteplirsen to Europe.”


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