OncLive’s September EMA Regulatory Recap: Key EU Approvals in Oncology

Below is your guide to the regulatory milestones that shaped cancer care in the European Union (EU) in September, featuring key approvals by the European Commission (EC), as well as critical recommendations and validations from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

These decisions introduced novel therapeutic indications across several malignancies, including rare tumors and gliomas, advanced companion diagnostics, and introduced patient-friendly subcutaneous formulations of effective agents. Read on to learn more!

What Were the Key EU Approvals of September 2025?

9/18: European Commission Green-Lights Vimseltinib for Symptomatic, Unresectable TGCT

The EC granted approval to vimseltinib (Romvimza) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT).1 Patients’ disease must be associated with clinically relevant physical function deterioration, and surgical options must either be exhausted or determined likely to induce unacceptable morbidity or disability.

The regulatory decision was supported by efficacy and safety findings derived from 2 key studies: the phase 3 MOTION trial (NCT05059262) and a phase 1/2 trial (NCT03069469). MOTION evaluated vimseltinib in patients with TGCT who were not suitable candidates for surgery and who had not received prior anti-CSF1/CSF1R therapy, whereas the phase 1/2 study evaluated the safety, efficacy, and pharmacokinetics of vimseltinib for the treatment of patients with malignant solid tumors and TGCT.1,2

In a descriptive analysis from MOTION conducted at week 97, 23% of patients (n = 19 of 83) who were randomly assigned to receive vimseltinib achieved the best overall response of complete response (CR).1 The efficacy was determined per RECIST 1.1 criteria via blinded independent radiological review. The analysis established that the median time to CR was 11.5 months. The safety profile observed for vimseltinib during these trials was characterized as manageable and consistent with previously generated data from the phase 1/2 study.

9/22: Vorasidenib Nets European Approval in IDH1/2-Mutated Grade 2 Glioma

Vorasidenib (Voranigo) was approved by the EC for adult and adolescent patients 12 years of age and older who weigh at least 40 kg and present with predominantly non-enhancing grade 2 astrocytoma or oligodendroglioma and harbor an IDH1 R132 or IDH2 R172 mutation.3 Treatment is limited to those who have only undergone surgical intervention and are not in immediate need of receiving radiotherapy or chemotherapy.

Data from the pivotal phase 3 INDIGO trial (NCT04164901) informed this regulatory decision. Findings published in The New England Journal of Medicine showed that vorasidenib met the study’s primary end point of improved progression-free survival (PFS) vs placebo.4 At a median follow-up of 14.0 months (interquartile range, 10.1-17.9), patients who were treated with vorasidenib (n = 168) achieved a median PFS of 27.7 months (95% CI, 17.0-not estimable [NE]) vs 11.1 months (95% CI, 11.0-13.7) among those who received placebo (n = 163). This result was statistically significant, as evidenced by a HR of 0.39 (95% CI, 0.27-0.56; P < .001).

What Were Some Other Regulatory Decisions of Interest?

9/5: Ventana Assay Earns CE IVDR Approval in EU as Companion Diagnostic in HER2-Expressing Breast and Biliary Tract Cancers

The VENTANA HER2 (4B5) Rabbit Monoclonal Primary Antibody RxDx assay received CE IVDR approval for 2 distinct label expansions within the EU.5

The first expansion indicated that the assay can now identify patients with hormone receptor–positive metastatic breast cancer whose disease is also categorized as HER2-ultralow. This identification is crucial for determining patient eligibility for treatment using fam-trastuzumab deruxtecan-nxki (T-DXd; Enhertu).

The second expansion categorized the assay as a companion diagnostic in patients with biliary tract cancer. It is now approved to aid in assessing HER2-positivity status and a HER2 immunohistochemistry (IHC) score of 3+ in this population. Patients with positive assay results will be eligible for treatment with zanidatamab-hrii (Ziihera).

Validation of the Ventana assay was conducted during the phase 3 DESTINY-Breast06 trial (NCT04494425), which compared T-DXd against the investigator’s choice of chemotherapy in patients with hormone receptor–positive, HER2-low or -ultralow metastatic breast cancer who had received 1 or more lines of endocrine therapy but no previous chemotherapy in the metastatic setting.6 The Ventana assay was utilized centrally to determine the HER2 status for patients screened for DESTINY-Breast06.

9/11: EMA Validates Marketing Authorization Application for T-DXd in HER2+ Metastatic Solid Tumors

The EMA validated a type II variation marketing authorization application for T-DXd, initiating the formal scientific review process by the EMA’s CHMP.7 The application is seeking approval for T-DXd in treating adult patients with HER2-positive (IHC 3+) unresectable or metastatic solid tumors who have received prior treatment and have no satisfactory alternative treatment options available.

The application is supported by clinical data gathered from 3 separate phase 2 trials, all demonstrating clinically meaningful responses across patients with HER2-positive solid tumors. These trials include:

• DESTINY-PanTumor02 (NCT04482309) in patients with various solid tumors, such as pancreatic, biliary tract, bladder, ovarian, cervical, and endometrial
• DESTINY-CRC02 (NCT04744831) in patients with colorectal cancer
• DESTINY-Lung01 (NCT03505710) in patients with non–small cell lung cancer (NSCLC)

If approved, T-DXd would be the first antibody-drug conjugate and the first HER2-targeted therapy to receive a tumor-agnostic indication in the EU.

What Potentials Approvals Are Coming Down the Pike?

9/18: CHMP Recommends EU Approval of Subcutaneous Mosunetuzumab in R/R Follicular Lymphoma

The EMA’s CHMP also recommended the approval of a SC formulation of mosunetuzumab (Lunsumio) for the treatment of adult patients who have relapsed/refractory follicular lymphoma following at least 2 prior lines of systemic therapy.8

The opinion is supported by data generated during the primary analysis of the phase 1/2 GO29781 study (NCT02500407), which confirmed the efficacy of the SC formulation in this population. Patients who received SC mosunetuzumab achieved a high overall response rate of 74.5% (95% CI, 64.4%-82.9%) and a CR rate of 58.5% (95% CI, 47.9%-68.6%) per independent review committee assessment. Furthermore, the median duration of CR was noted as 20.8 months (95% CI, 18.8-NE). An important pharmacokinetic finding confirmed that the SC formulation achieved noninferiority when compared with the available intravenous (IV) administration of mosunetuzumab.

The EC is expected to issue its final decision regarding the marketing authorization in the near future.

9/19: Pembrolizumab Earns 2 Positive CHMP Opinions in HNSCC, Other EU Indications

The EMA’s CHMP issued 2 positive opinions for pembrolizumab (Keytruda).9 These include:

• The approval of a novel formulation of pembrolizumab for SC injection across all adult indications in the EU
• A new indication for pembrolizumab as part of a perioperative regimen for the treatment of select adult patients with resectable locally advanced head and neck squamous cell carcinoma (HNSCC).

The marketing authorization application for the SC formulation was supported by results from the pivotal, open-label phase 3 3475A-D77 trial (NCT05722015) comparing the SC vs IV formulations of pembrolizumab in adult patients with newly diagnosed stage IV metastatic NSCLC (squamous or nonsquamous) who did not possess sensitizing EGFR, ALK, or ROS1 alterations.

The basis for the HNSCC recommendation comes from data from the phase 3 KEYNOTE-689 trial (NCT03765918), an open-label study evaluating perioperative pembrolizumab followed by standard surgery and adjuvant radiotherapy (with or without cisplatin) vs the standard of care alone.

Both positive opinions for pembrolizumab will now proceed to review by the EC or final marketing authorization across the EU, Iceland, Liechtenstein, and Norway. Final decisions on these authorizations are anticipated in the fourth quarter of 2025.

References

1. Deciphera receives European Commission approval of Romvimza (vimseltinib) for the treatment of tenosynovial giant cell tumor (TGCT). News release. Deciphera. September 17, 2025. Accessed October 2, 2025. https://www.deciphera.com/news/deciphera-receives-european-commission-approval-for-romvimza
2. Study of vimseltinib (DCC-3014) in patients with advanced tumors and tenosynovial giant cell tumor. ClinicalTrials.gov. Updated November 20, 2024. Accessed September 18, 2025. https://www.clinicaltrials.gov/study/NCT03069469
3. European Commission approves Servier’s Voranigo (vorasidenib) as the first targeted therapy for grade 2 IDH-mutant glioma in the EU. News release. Servier. September 22, 2025. Accessed October 2, 2025. https://www.prnewswire.com/news-releases/european-commission-approves-serviers-voranigo-vorasidenib-as-the-first-targeted-therapy-for-grade-2-idh-mutant-glioma-in-the-eu-302562180.html
4. Mellinghoff IK, van den Bent MJ, Blumenthal DT, et al. Vorasidenib in IDH1- or IDH2-mutant low-grade glioma. N Engl J Med. 2023;389(7):589-601. doi:10.1056/NEJMoa2304194
5. Roche receives CE IVDR approval for HER2 (4B5) companion diagnostic test to identify HER2-ultralow breast cancer and biliary tract cancer patients. News release. Roche Diagnostics. September 5, 2025. Accessed October 2, 2025. https://diagnostics.roche.com/global/en/news-listing/2025/roche-receives-ce-ivdr-approval-for-her2--4b5--companion-diagnos.html
6. Shami R, Salgado R, Bardia A, et al. Analytical and clinical validation of PATHWAY HER2 (4B5) assay for assessment of HER2-low/HER2-ultralow status and eligibility for trastuzumab deruxtecan in DESTINY-Breast06. ESMO Open. 2025;10(6):105310. doi:10.1016/j.esmoop.2025.105310
7. Enhertu type II variation application validated in the EU for previously treated patients with HER2 positive metastatic solid tumors. News release. Daiichi-Sankyo. September 11, 2025. Accessed October 2, 2025. https://www.daiichisankyo.com/files/news/pressrelease/pdf/202509/20250911_E.pdf
8. CHMP recommends EU approval of Roche’s subcutaneous formulation of Lunsumio for people with relapsed or refractory follicular lymphoma. News release. Roche. September 18, 2025. Accessed October 2, 2025. https://www.roche.com/media/releases/med-cor-2025-09-19
9. Merck receives two positive EU CHMP opinions for KEYTRUDA (pembrolizumab), for subcutaneous (SC) administration and for new indication for earlier-stage head and neck cancer. News release. Merck. September 19, 2025. Accessed October 2, 2025. https://www.merck.com/news/merck-receives-two-positive-eu-chmp-opinions-for-keytruda-pembrolizumab-for-subcutaneous-sc-administration-and-for-new-indication-for-earlier-stage-head-and-neck-cancer/