This page shows the latest Exondys 51 news and features for those working in and with pharma, biotech and healthcare.
Vyondys 53 is Sarepta’s second approved DMD therapy after Exondys 51 (eteplirsen).
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
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 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
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
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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Alnylam can take heart in exceptions, however, such as Sarepta, with its Duchenne treatment Exondys 51, and hope it too can buck this trend.
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
Licence. 145. Sarepta Therapeutics/ Gilead Sciences. PRV received when EXONDYS 51 was FDA approved for Duchenne muscular dystrophy.
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