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FDA Grants Fast-Track Approval to Enhertu for Treatment of HER2+ Solid Tumors


5 April 2024

The US FDA has granted accelerated approval to ENHERTU®, the first tumor-agnostic, HER2-directed therapy developed by Daiichi Sankyo and AstraZeneca for metastatic HER2-positive solid tumors. The approval is based on significant clinical responses observed across various cancer types, with ongoing reviews by international regulatory partners under Project Orbis.

Daiichi Sankyo and AstraZeneca have announced that the US Food and Drug Administration (FDA) has granted accelerated approval to ENHERTU® (fam-trastuzumab deruxtecan-nxki) for the treatment of metastatic HER2-positive solid tumors. This makes ENHERTU® the first tumor-agnostic, HER2-directed therapy, offering new hope to patients with previously treated metastatic cancers that express high levels of HER2 (immunohistochemistry [IHC] 3+), who previously had no satisfactory alternative treatment options.

This approval was underpinned by the compelling outcomes of three phase 2 trials encompassing a diverse range of solid tumors. A total of 192 adult patients, previously treated for unresectable or metastatic HER2-positive solid tumors, participated across the DESTINY-PanTumor02, DESTINY-Lung01, and DESTINY-CRC02 trials.

Specifically, the DESTINY-PanTumor02 trial, which included patients with a variety of solid tumors such as biliary tract, bladder, and ovarian cancers, showed a confirmed ORR of 51.4% and a median DOR of 19.4 months. The DESTINY-Lung01 trial focused on non-small cell lung cancer (NSCLC), revealing a confirmed ORR of 52.9% and a median DOR of 6.9 months. Meanwhile, the DESTINY-CRC02 trial, which evaluated patients with colorectal cancer, reported a confirmed ORR of 46.9% and a median DOR of 5.5 months.

Dr. Funda Meric-Bernstam of The University of Texas MD Anderson Cancer Center emphasized the importance of this approval, highlighting the limited treatment options previously available to patients with metastatic HER2 positive solid tumors. She stated, “Based on the clinically meaningful response rates seen across clinical trials, this tumor-agnostic approval means that patients may now be treated with a HER2 directed medicine.”

The FDA's decision to approve ENHERTU® under its Real-Time Oncology Review (RTOR) program, with Priority Review and Breakthrough Therapy Designation, underscores the urgent need for effective treatments for this patient population. Moreover, the review was part of Project Orbis, allowing for concurrent submission and review among international regulatory partners, indicating a global effort in advancing cancer treatment.

“As the first antibody drug conjugate to be granted a tumor agnostic indication, ENHERTU is truly delivering on its potential across metastatic HER2 targetable tumors. This approval also elevates the importance of testing for biomarkers, including HER2, across a broad range of tumors to ensure these patients with advanced cancer who have few options know whether a targeted medicine might be right for them,” said Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca.

ENHERTU®'s safety profile was thoroughly evaluated in 347 adult patients across the aforementioned trials. The treatment was associated with a range of adverse reactions, the most common of which included issues related to blood cell counts, fatigue, and gastrointestinal disturbances.


ENHERTU, a leading HER2-directed antibody-drug conjugate (ADC) developed using Daiichi Sankyo’s proprietary DXd ADC Technology, is a pivotal part of both Daiichi Sankyo's and AstraZeneca's oncology portfolios. It combines a HER2 monoclonal antibody with a topoisomerase I inhibitor payload, demonstrating significant efficacy in various cancers. Approved in over 60 countries for specific types of HER2-positive breast cancer, and in more than 35 countries for certain HER2-mutant non-small cell lung cancers (NSCLC), ENHERTU's global acceptance is based on results from the DESTINY-Breast03 and DESTINY-Lung02 trials, with continued U.S. approval for NSCLC contingent on further trials.


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