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Sarepta Therapeutics was dealt a surprising setback Monday when the Food and Drug Administration rejected its marketing application for a second drug that aimed to treat children with Duchenne muscular dystrophy, a rare, inherited muscle-wasting disease.

In a statement, Sarepta said the FDA denied the approval of its drug, called Vyondys 53, due to the risk of infections related to intravenous infusion ports and kidney toxicity seen in animal experiments.

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  • My two son is duchenne Muscular dystrophy (DMD) patient, error exon 53. 1-Ayush Tiwari 13y old. 2-Harsh Tiwari 8y old. I live in INDIA.

  • This is all due to hubris. Management should have OVER-invested in this trial since 2016. Not only did they fail to do that, they also failed to provide the follow up data FDA requested since Exondys 51 was approved almost 4 years ago. They have a very conditional approval for Exondys 51, and who knows what the implications are for that drug? They’ve put their entire franchise in jeopardy.

    This is a terrible outcome for patients and their families. Really tragic because this company, over the last four years, could have built a champion for rare diseases. Instead management is unlikely to be able to dig itself out of its pipeline hole.

    • The terrible outcome for patients and taxpayers happened when the FDA approved Exondys 51. There is a reason Sarepta hasn’t completed the required studies; the drug, very likely, doesn’t work. It never did.

  • Much was made of the twins with DMD that kept walking while treated with Exondys 51. What happened to them?

    • My son has DMD and we were waiting for Vyondys 53 to be approved. Looks like it’s not going to happen anytime soon. Nitin, you could contact me if you would like to discuss.

  • ACER, Chiesi’s recent CRL on CF, the weird ZGNX pushback, the bizarre approval of Ruzurgi contra to the orphan drug act. There’s no doubt there is a policy change at the FDA, just not sure the FDA knows what it is yet

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