A Shift In Thinking: FDA Moves Toward Easier Neurofibromatosis Endpoints
Volumetric imaging gains favor as a primary endpoint, along with single-arm studies, potentially creating a faster path to drug approval in the rare disease.
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FDA’s patient-focused drug development initiative and its Drug Development Tool for qualifying endpoints are galvanizing efforts to establish sarcopenia as an indication for clinical development in what could be a very significant market of elderly patients with weakness and functional disability.
Pink Sheet Podcast: Finally, A US FDA User Fee Deal, ‘Pandemic Is Over’ Take-Aways, Tpoxx Development Issues
Pink Sheet reporters and editor discuss the impact of the long wait for a user fee deal to be negotiated, how the FDA will interpret President Biden’s statement that the COVID-19 pandemic is over, and concerns with the clinical development of the monkeypox treatment Tpoxx.
US FDA's Vaccines and Related Biological Products Advisory Committee endorses Rebyota for recurrent C. difficile infection; most panelists prefer a product whose manufacturing will be regulated, even one with lingering concerns about efficacy or safety, over the current landscape.