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.
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.
Elevidys (delandistrogene moxeparvovec-rokl) gained an accelerated US Food and Drug Administration approval in June to treat ambulatory patients ages four...
Revised recommendation is based on most recent data on circulating strains in US, where KP.3 is now dominant. Novavax sticks with plans targeting the JN.1 ‘parent strain.’
With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.
Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.
Recent ODAC meetings suggest the FDA will expand focus beyond applications with pending approval decisions to refining trial designs and drug labels for competitors.
Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.
Recent ODAC meetings suggest the FDA will expand focus beyond applications with pending approval decisions to refining trial designs and drug labels for competitors.
The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.
