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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 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 –

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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

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

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
FDA rejects Sarepta’s new Duchenne drug over safety fears

Vyondys 53 (golodirsen) follows Exondys 51, the first and currently only disease-modifying treatment approved in the US for the inherited muscle-wasting disease. ... Sarepta gained approval for Exondys 51 three years ago amid controversy about its lack
FDA will maintain the pace, vows Woodcock, but warns of skills gap

These cases have, of course, not been without controversy – one of the most notable being the approval of Sarepta’s Duchenne treatment Exondys 51 in 2016, despite questionable data.

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Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe

Alnylam can take heart in exceptions, however, such as Sarepta, with its Duchenne treatment Exondys 51, and hope it too can buck this trend.
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

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

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