FDA Grants Accelerated Approval for Trastuzumab Deruxtecan, Recognizing HER2+ as a Tumor-Agnostic Biomarker

The US FDA granted accelerated approval to trastuzumab deruxtecan-nxk (Enhertu) on April 5, 2024, for the treatment of adult patients with unresectable or metastatic HER2-positive solid tumors who have received prior systemic treatment and have no satisfactory alternative options. This approval is noteworthy as HER2-positive status has become a tumor-agnostic biomarker, allowing for a broader application across various cancers​ 

The approval was based on data from three pivotal clinical trials: DESTINY-PanTumor02 (NCT04482309), DESTINY-Lung01 (NCT03505710), and DESTINY-CRC02 (NCT04744831). These trials demonstrated significant efficacy in previously treated patients. The objective response rates (ORRs) were 51.4%, 52.9%, and 46.9%, respectively, with median durations of response (DOR) ranging from 5.5 to 19.4 months across the studies​ 

This decision underscores the growing importance of HER2-positive as a predictive biomarker beyond breast and gastric cancers, potentially transforming therapeutic strategies for diverse HER2-expressing tumors. The ongoing monitoring of clinical benefit in confirmatory trials will be crucial for continued approval​.

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Related article: https://www.actgenomics.com/insights-blog/destiny-pantumor02-the-adc-trastuzumab-deruxtecan-t-dxd-showed-activity-in-her2-expressing-solid-tumors

References:

1. Meric-Bernstam F et al., J Clin Oncol. 2024;42(1):47-58.
2. Smit EF et al., Lancet Oncol. 2024;25(4):439-454.
3. Raghav KPS et al., J Clin Oncol. 2023;41(suppl 16).

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