The European Medicines Agency (EMA) has validated Daiichi Sanyoko‘s marketing authorization application for trastuzumab deruxtecan (DS-8201) for the treatment of inoperable or metastatic HER2-positive breast cancer, the company announced.
The validation means that the application is complete and may now begin the scientific review process by the Committee for Medicinal Products for Human Use. The committee also granted trastuzumab deruxtecan accelerated assessment, as a therapeutic innovation expected to be of major public health interest.
Accelerated assessment status can significantly reduce bureaucratic review times.
Developed in collaboration with AstraZeneca, trastuzumab deruxtecan is an antibody-drug conjugate that specifically targets cancer cells that carry the HER2 protein. It consists of a HER2-specific antibody and the cancer-killing agent deruxtecan, a derivative of exatecan. It is a potential treatment for all types of HER2-positive cancers.
“The accelerated assessment highlights the significant unmet need for patients with HER2 positive metastatic breast cancer that trastuzumab deruxtecan aims at addressing,” said Gilles Gallant, PhD, global head of oncology R&D at Daiichi Sankyo, in a press release. “Trastuzumab deruxtecan is already available for patients in the U.S. and Japan, and we look forward to working with the EMA to bring this important new medicine to patients in the EU as quickly as possible.”
The U.S. Food and Drug Administration had granted trastuzumab deruxtecan accelerated approval under the trade name Enhertu earlier this year. Accelerated approval is a type of conditional approval granted to medications that fill an unmet medical need in a serious medical condition.
Enhertu’s full approval will depend on further verification of its clinical benefit in a confirmatory study.
The EMA based its current decision on positive results from the Phase 2 DESTINY-Breast01 trial (NCT03248492). The trial assessed the safety and efficacy of trastuzumab deruxtecan at a dose of 5.4 mg per kilogram of body weight in 253 patients with inoperable and/or metastatic HER2-positive tumors. All participants had also received at least two prior HER2 therapy regimens, including the antibody-drug conjugate Kadcycla (ado-trastuzumab emtansine).
The trial’s primary outcome, or main therapeutic goal, was the percentage of participants to achieve either a complete response, meaning that their cancer disappeared, or a partial response, meaning that their tumors shrank significantly.
Results from the first 184 patients given Enhertu in the trial, of which 112 (60.9%) responded favorably to treatment, have been published in The New England Journal of Medicine. These included 103 (56%) who achieved a partial response and eight (4.3%) who achieved a complete response.
Daiichi Sankyo has also submitted a supplemental new drug application for trastuzumab deruxtecan in Japan.