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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.

Lilly links up with Precision BioSciences in genome editing deal

Lilly links up with Precision BioSciences in genome editing deal

Vyondys 53 is Sarepta’s second approved DMD therapy after Exondys 51 (eteplirsen).

Latest news

  • FDA approves Sarepta rival NS Pharma’s Duchenne drug FDA approves Sarepta rival NS Pharma’s Duchenne drug

    This represents around 8% of the overall DMD population, while those with the exon 51 mutation account for around 13%. ... It is the company’s second DMD drug after Exondys 51 (eteplirsen), the first disease-modifying treatment for the disease to be

  • FDA sets Q3 decision date for NS Pharma’s DMD drug FDA sets Q3 decision date for NS Pharma’s DMD drug

    However, after an appeal of the decision, Sarepta was granted an approval for its second DMD medicine – after its exon 51 targeting Exondys 51 – the FDA deemed that the drug has ... NS Pharma could price its drug below Sarepta’s to gain an edge –

  • Wave’s challenge to Sarepta in Duchenne falls flat Wave’s challenge to Sarepta in Duchenne falls flat

    Wave will abandon a phase 2/3 trial of suvodirsen called DYSTANCE 51, ending any hope of filing for accelerated approval of the drug as a potential rival to Sarepta’s  ... Exondys 51 (eteplirsen), which was approved three years ago and made around

  • FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection

    Vyondys uses the same ‘exon skipping’ mechanism, but helps patients with the exon 53 mutation rather than the exon 51 mutation targeted by Exondys 51. ... Sarepta has indicated it will launch Vyondys 53 at a price that is “at parity” with Exondys

  • Sarepta rival NS Pharma files Duchenne drug with FDA Sarepta rival NS Pharma files Duchenne drug with FDA

    The treatment is a follow-up to Sarpeta’s Exondys 51, the first and currently only disease-modifying treatment approved in the US for the inherited muscle-wasting disease. ... Vyondys uses the same ‘exon skipping’ mechanism, but helps patients with

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