Sarepta Therapeutics is to seek full approval for its Duchenne gene therapy Elevidys in the US and partners Roche have likewise vowed to seek approval in ex-US countries, despite the product failing to hit the primary endpoint of its Phase III study.
Sarepta And Roche Press Ahead With Elevidys Filing Despite Duchenne Trial Miss
The Phase III EMBARK study has fallen short on its primary endpoint – but Sarepta believes the trial has otherwise produced robust data, and believes a full US approval is justified.
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Pharma executives and investors are waiting with bated breath to find out if President Trump will include drugs in a new round of tariffs to be announced on 2 April.
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One CDRH employee said the cuts already are having a major effect on morale.
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Mass FDA layoffs on 1 April were designed to spare product reviewers, but still touched many who are critical to the application review process or drug development, which could mean fewer treatments are brought to the US market first.
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Experts say PBMs should become familiar with transparency rules and prepare to release data, although more legal wrangling could occur.
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Optum Rx, one of the largest PBMs in the US, declared it was the “first comprehensive, transparent pharmacy services company” after announcing several consumer-friendly changes.
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FDA Commissioner Martin Makary was sworn in Friday and knew of the plan that ultimately lead to CBER director Peter Marks' resignation on 28 March.
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In his resignation letter, the CBER director said he was willing to work with HHS Secretary Robert F. Kennedy Jr. to address concerns about vaccine safety, but "it has become clear that truth and transparency are not desired by the secretary."