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Exondys 51

This page shows the latest Exondys 51 news and features for those working in and with pharma, biotech and healthcare.

FDA reviewer unimpressed with PTC’s Translarna

FDA reviewer unimpressed with PTC’s Translarna

that the firm was emboldened by the FDA’s approval last year of another DMD therapy from Sarepta - Exondys 51 (eteplirsen) - after Janet Woodcock, head of the agency’s Center for

Latest news

  • Sarepta eyes FDA filing for DMD drug golodirsen after positive trial Sarepta eyes FDA filing for DMD drug golodirsen after positive trial

    The exon-skipping drug raised functional dystrophin levels. Sarepta is preparing to file a follow-up to its Duchenne muscular dystrophy (DMD) treatment Exondys 51 that seems to have greater efficacy. ... normal from an average baseline level of 0.095% of

  • FDA approves first steroid for Duchenne muscular dystrophy FDA approves first steroid for Duchenne muscular dystrophy

    The agency granted orphan and priority review status for the drug, which is the second DMD treatment to be approved in the US after Sarepta Therapeutics' exon-skipping therapy Exondys 51 ... Marathon's chief executive Timothy Cunniff, alluding to the

  • A rock and a hard place A rock and a hard place

    Last month the FDA approved Sarepta's Duchenne muscular dystrophy (DMD) drug known as Exondys 51. ... Remember that some members of the FDA committee reviewing the data on Exondys 51 thought the drug should be approved.

  • Sarepta soars as FDA clears its muscular dystrophy drug Sarepta soars as FDA clears its muscular dystrophy drug

    Sarepta shares closed up 74% yesterday after news of the conditional approval for Exondys 51 (eteplirsen) broke - taking investors by surprise. ... Exondys 51 is indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon

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Latest Intelligence

  • Europe vs the US: New drug product approvals Europe vs the US: New drug product approvals

    Among the noteworthy orphan approvals are Exondys 51 (for the treatment of Duchenne muscular dystrophy, only approved in the US), Darzalex (for the treatment of multiple myeloma, approved in the US

  • Deal Watch February 2017 Deal Watch February 2017

    Licence. 145. Sarepta Therapeutics/ Gilead Sciences. PRV received when EXONDYS 51 was FDA approved for Duchenne muscular dystrophy.

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