Exondys 51

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

Duchenne UK backs Evox dystrophin delivery project

At the moment that includes already-market drugs that skip over or ignore the mutation in the dystrophin gene – such as PTC Therapeutics’ Translarna (ataluren) and Sarepta’s Exondys 51 (eteplirsen) –

Latest news

CHMP backs Spark’s Luxturna, but rejects Sarepta again

Eteplirsen was approved in the US as Exondys 51 in 2016, although the decision came after FDA reviewers recommended that the drug be rejected as the evidence for its benefits was ... which there was no meaningful difference between Exondys and placebo in
Pfizer abandons antibody for Duchenne

News of the setback spurred modest gains in the share price of Sarepta, which sells exon-skipping therapy Exondys 51 (eteplirsen) in the US and Europe, as well as PTC Therapeutics
Daily Brief: Sarepta deepens gene therapy investment, NICE changes mind on Besponsa, Digital Medicine company Akili raises $13m

Sarepta has made its name by launching the first ever FDA approved treatment for Duchenne muscular dystrophy, Exondys 51, and is now looking to expand its pipeline into other rare disease ... Sarepta has also just announced rising sales of Exondys 51,
Mallinckrodt doses first patient in muscular dystrophy trial

For now, the only approved therapies are Sarepta’s exon-skipping therapy Exondys 51 (eteplirsen), which has some controversy over its efficacy, PTC’s Translarna (ataluren), which is available in Europe
Summit plunges after Duchenne disappointment

Sarepta's investment in the field has been possible thanks to the FDA’s approval of the first US-approved Duchenne drug, Exondys 51, in 2016, despite controversies over its efficacy. ... The field is marked by the curiosity of the EMA having approved

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Latest Intelligence

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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