Regulatory & Policy

CHMP backs tumor-agnostic EU use of Enhertu across HER2-positive solid tumors

Daiichi Sankyo and AstraZeneca's Enhertu receives CHMP recommendation for HER2-positive solid tumors in the EU

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending Enhertu (trastuzumab deruxtecan) for approval in the EU as a monotherapy for adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior treatment and have no satisfactory treatment options, according to an announcement from Daiichi Sankyo. The recommendation covers a tumor-agnostic population, potentially making Enhertu the first HER2-directed therapy approved for a tumor-agnostic indication in the EU.

The CHMP opinion will now be reviewed by the European Commission, which holds the authority to grant marketing authorizations across EU member states. Daiichi Sankyo (TSE: 4568), headquartered in Tokyo, and its collaborator AstraZeneca (LSE/STO/NYSE: AZN) are jointly developing and commercializing the asset.

Clinical evidence supporting the recommendation

The CHMP based its positive opinion on data from three Phase II trials: DESTINY-PanTumor02, DESTINY-Lung01, and DESTINY-CRC02, each evaluating Enhertu at 5.4 mg/kg in previously treated patients with centrally or locally confirmed HER2-positive tumors.

In DESTINY-PanTumor02, a global, multicenter, open-label study enrolling patients with biliary tract, bladder, cervical, endometrial, ovarian, pancreatic, or other tumors, Enhertu produced a confirmed objective response rate (ORR) of 51.4% (95% CI: 41.7–61.0) and a median duration of response (DOR) of 14.2 months in 111 HER2-positive (IHC 3+) patients. In DESTINY-Lung01, which evaluated the drug in HER2-overexpressing non-small cell lung cancer, a confirmed ORR of 52.9% (95% CI: 27.8–77.0) and a median DOR of 6.9 months were observed in 17 IHC 3+ patients. In DESTINY-CRC02, a randomized two-arm Phase II trial in HER2-positive colorectal cancer, the confirmed ORR was 46.9% (95% CI: 34.3–59.8) with a median DOR of 5.5 months among 64 IHC 3+ patients.

The safety profile across the three trials was described by the company as consistent with prior Enhertu clinical experience. In DESTINY-PanTumor02, the most common grade 3 or higher drug-related adverse events were neutropenia and anemia, each occurring in 10.9% of patients. Interstitial lung disease or pneumonitis, a recognized risk with this drug class, was observed in 10.5% of patients, with three grade 5 events reported. Rates were lower in DESTINY-CRC02, where all ILD or pneumonitis events were grade 1 or 2.

Scientific and competitive context

HER2 overexpression has been a validated therapeutic target in breast, gastric, and salivary gland cancers for over two decades, but its role across a broader range of solid tumors has only recently been systematically explored. In tumor types such as biliary tract, colorectal, cervical, and pancreatic cancer, HER2 testing is not routinely performed in the EU, and no HER2-directed therapies have previously been authorized for these indications in that region.

The CHMP recommendation positions Enhertu within a tumor-agnostic framework, a relatively recent regulatory approach that defines eligibility by molecular marker rather than tissue of origin. In the US, the FDA granted accelerated approval to Enhertu for HER2-positive solid tumors in 2024, and the drug has since received approval in more than 15 countries for this indication. The EU recommendation, if converted to a full marketing authorization by the European Commission, would extend that reach to one of the largest regulated pharmaceutical markets globally.

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Enhertu is a HER2-directed antibody drug conjugate built on Daiichi Sankyo's proprietary DXd platform, in which a HER2-targeting monoclonal antibody is linked to a topoisomerase I inhibitor payload via a cleavable tetrapeptide linker. The high drug-to-antibody ratio and membrane-permeable payload are thought to contribute to activity in tumors with lower HER2 expression levels, though the current recommendation is limited to IHC 3+ patients. The drug's established presence across breast, gastric, and lung cancer settings gives it a broad clinical footprint against which this tumor-agnostic application adds a further dimension.

No directly competing HER2-directed tumor-agnostic therapy has received EU authorization to date, though the ADC landscape is expanding, with multiple assets in development targeting HER2 and other receptors across solid tumor types.

Enhertu's broader development program

The Daiichi Sankyo and AstraZeneca collaboration, initiated in 2019, has built Enhertu into one of the most extensively studied ADCs in oncology. The drug currently holds approvals in more than 95 countries for HER2-positive and HER2-low breast cancer, more than 75 countries for HR-positive, HER2-low or HER2-ultralow breast cancer, more than 70 countries for HER2-mutant NSCLC, and more than 90 countries for HER2-positive gastric or gastroesophageal junction adenocarcinoma. Recent approvals in breast cancer have also extended to neoadjuvant and first-line settings based on the DESTINY-Breast11 and DESTINY-Breast09 trials, respectively.

The tumor-agnostic indication adds to a growing field of biomarker-selected, histology-independent approvals, a regulatory pathway that has gained traction with agents targeting NTRK fusions, MSI-H tumors, and BRAF V600E mutations. Whether HER2 IHC 3+ status achieves similar cross-tumor regulatory acceptance in the EU will depend on the European Commission's review of the CHMP opinion. A timeline for that decision was not specified in the company's announcement.


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