Ultragenyx CEO: FDA Leaders, Review Divisions Out Of Sync On Accelerated Approval For Gene Therapy
Executive Summary
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.
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