Sarepta got a ‘surprise’ rejection

The FDA yesterday rejected Sarepta Therapeutics’ second proposed therapy for Duchenne muscular dystrophy, a decision that left the company’s CEO “very surprised.”

As STAT’s Adam Feuerstein reports, the FDA’s stated reasons were the risk of infections related to intravenous infusion ports and kidney toxicity seen in animal experiments, according to Sarepta. None of that came up during the agency’s review, according to Doug Ingram, Sarepta’s CEO.

The rejection only underlines outstanding questions about Sarepta’s relationship with the FDA, dating back to a controversial 2016 approval that divided agency scientists.

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