Individual Endpoints Pose Challenge For US FDA In Review Of Rare Disease Drug
FDA reviewer describes difficulties in reviewing application for Mepsevii for Sly syndrome; the experience provides perspective for other companies seeking to use novel endpoints.
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In a Pink Sheet interview Emil Kakkis contends the agency’s recruitment challenges, high turnover rates and a lack of expertise on the diseases for which reviewers are evaluating drugs have created a disconnect between FDA leadership’s stated willingness to permit accelerated approval for gene therapy and what review divisions are doing on the ground.
Formal reg not needed, according to agency; officials agree that regulatory flexibility is best way to offer sponsors advice, saying data collected during compassionate use can be helpful in guiding regulatory decisions.
The latest drug approval and development news from our US FDA Performance Tracker.