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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
Vyondys 53 is Sarepta’s second approved DMD therapy after Exondys 51 (eteplirsen).
Latest news
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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
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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
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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
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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 approves Sarepta rival NS Pharma’s Duchenne drug
FDA sets Q3 decision date for NS Pharma’s DMD drug
Wave’s challenge to Sarepta in Duchenne falls flat
FDA grants surprise OK to Sarepta’s DMD therapy after earlier rejection
Sarepta rival NS Pharma files Duchenne drug with FDA
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Approximately 3 fully matching, plus 18 partially matching documents found.
Latest Intelligence
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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.
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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
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Deal Watch February 2017
Licence. 145. Sarepta Therapeutics/ Gilead Sciences. PRV received when EXONDYS 51 was FDA approved for Duchenne muscular dystrophy.
Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe
Europe vs the US: New drug product approvals
Deal Watch February 2017
More from intelligence
Approximately 0 fully matching, plus 3 partially matching documents found.
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