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Alexander Drilon, MD

Gregory J. Riely, MD, PhD


FDA Brief: New Guidance for Industry on Tissue-Agnostic Drug Development

By: Lauren Velentzas
Posted: Tuesday, November 22, 2022

The U.S. Food and Drug Adminstration (FDA) recently released draft guidance on tissue-agnostic drug development in oncology. When finalized, this draft guidance may further facilitate the approval of future treatments across multiple cancer types targeting specific molecular alterations such as NTRK gene fusions.

According to the 16-page document released by the FDA, “tissue-agnostic oncology drug development may represent a more feasible developmental strategy” for rare tumor types such as those driven by NTRK gene fusions. Among other recommendations, the FDA suggests sponsors consider clinical pharmacology, safety and efficacy profiles, and “whether a drug could be developed more efficiently in that cancer type rather than in a tissue-agnostic setting.”

Additionally, the draft provides information on how tissue-agnostic drugs can differ from more traditional cancer treatments and how this may influence the drug development process. There is an inherent need in tissue-agnostic drug development to generalize treatment effects based on the data, since they are mutation-based rather than tissue-based. As mentioned in the draft, “sponsors should have early and frequent discussions with [the] FDA to discuss development approaches that are critical to tissue-agnostic oncology drug development, including nonclinical data, justification for the sample sizes for the overall population and for subgroups of specific cancer types, and [the] approval pathway (traditional or accelerated approval).”

The draft follows the recent approval in September of the tissue-agnostic agent selpercatinib in the treatment of solid tumors with RET gene fusions. Two tissue-agnostic TRK inhibitors have been approved for cancers driven by NTRK gene fusions: larotrectinib in 2018 and entrectinib in 2019.

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