Ultragenyx CEO: FDA Leaders, Review Divisions Out Of Sync On Accelerated Approval For Gene Therapy
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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Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.
Quick takes on Ultragenyx’s CEO lobbing the US FDA for rare disease policy changes, the agency naming a new deputy assistant commissioner for media affairs, the former CEO of AAM starting a consulting firm, and more.
Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.