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FDA Can’t Find Efficacy For BioMarin’s Drisapersen

 
• By Derrick Gingery

On verge of advisory committee meeting, FDA says development program was exemplary, but is disappointed data are inconclusive.

Perhaps the most telling aspect of FDA's review of BioMarin Pharmaceutical Inc.’s proposed Duchenne muscular dystrophy treatment drisapersen is that no benefit-risk assessment was completed because staff found no benefit.

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US FDA Commissioner’s Office Plans Involvement In Many Approvals In Potential Major Change

 
• By Sarah Karlin-Smith

FDA Commissioner Martin Makary's remarks that his special assistant Tracy Beth Høeg will "be involved in other approval procedures" besides Novavax's COVID-19 vaccine application could be a dramatic change in operations, potentially making the approval system unpredictable.

Another Regeneron CRL Prompts Regulatory Operations Questions

 
• By Alaric DeArment

CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.

Canada’s HTA Agency Reveals How To Submit AI-Driven Evidence

 
• By Neena Brizmohun

Recognizing that the evidence it receives in applications for health technology assessments will increasingly be informed by artificial intelligence, the CDA-AMC has clarified its expectations for companies that use AI methods in the generation and/or reporting of evidence.