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.
You may also be interested in...
Supplemental Filings: Pink Sheet Notebook On Disease Lobbying, Dan Leonard’s Next Act, And More
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.
Ultra-Rare Orphan Drugs: Advocates Begin Laying Groundwork For New Incentive, Special Pathway
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.
Califf: Decades Of Follow-Up Necessary For Gene Therapy
FDA Commissioner Califf acknowledges that regulatory pathways for gene therapies are still being developed and that many factors outside of the modality impact agency requirements as he emphasized the need for long-term follow up due to the unknowns of gene modification.