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Sareptas Golodirsen snub highlights atypical communications, heralds higher bar at FDA- analysts

August 20, 2019 / Amirah Al Idrus

The FDA dealt a surprise rejection to Sarepta Therapeutics Monday, telling the company in a Complete Response Letter CRL that it was passing its second Duchenne muscular dystrophy DMD drug over due to risks of infusion port infection and kidney toxicity seen in animal studies. Sareptas first move will be to meet with the FDA to negotiate a black box warning or other labeling for the drug, golodirsen, to spell out the risk of kidney toxicity and infection risk, rather than to carry out more studies, wrote Evercore ISI analysts in a note Tuesday. But the implications of the rejection range beyond golodirsen. “The real driver(s) of the aytpical golodirsen CRL will continue to be a debate based on limited public disclosure of the granularity of facts, but the macro take home is clear in our minds—there is a regulatory “raising of the bar,” the analysts wrote. This could spell bad news for Sarepta’s other programs, namely its microdystrophin gene therapy that t...