August EMA Oncology
Approvals | Image Credit:
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Stay up to date with the latest regulatory milestones shaping cancer care across Europe. Below is your guide to all oncologic therapies approved by the European Commission (EC) in August 2025, including the pivotal clinical data behind each decision and expert perspectives on how these approvals are poised to impact real-world practice. Read on to learn more!

Nirogacestat (Ogsiveo) was approved by the EC for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.1 This decision marked a significant milestone, as there were previously no approved medicines in the European Union (EU) for patients with progressing desmoid tumors.

The regulatory decision was primarily supported by data from the phase 3 DeFi trial (NCT03785964). In this trial, patients treated with nirogacestat experienced a statistically significant improvement in progression-free survival (PFS) compared with those who received placebo (HR, 0.29; 95% CI, 0.15-0.55; P P

Patients also reported improvements in pain, desmoid tumor-specific symptoms, physical/role functioning, and overall health-related quality of life. The most common adverse effects (AEs) included diarrhea, rash, and ovarian toxicity in women of childbearing potential.

This approval follows the FDA’s approval of nirogacestat in November 2023 for the same indication, which was also supported by data from DeFi.2

The EC granted approval to the tablet formulation of zanubrutinib (Brukinsa) for use across all of its previously authorized indications.3

This regulatory decision follows the FDA’s approval of the tablet formulation in June 2025.4

Zanubrutinib is approved in the EU for several indications, including as monotherapy for adult patients with Waldenström macroglobulinemia, marginal zone lymphoma, and chronic lymphocytic leukemia, as well as in combination with obinutuzumab (Gazyva) for refractory/relapsed follicular lymphoma.5

The recommended dose of the tablet formulation is 320 mg per day, mirroring that of the capsule formulation, with patients now taking two 160 mg tablets daily.3

Safety data from compiled EU prescribing information for zanubrutinib monotherapy (n = 1550) show common AEs such as upper respiratory tract infection (36%), bruising (32%), hemorrhage/hematoma (30%), and neutropenia (30%).5 In the phase 2 ROSEWOOD trial (NCT03332017), which evaluated zanubrutinib plus obinutuzumab in relapsed/refractory follicular lymphoma, common AEs among patients treated with the combination (n = 143) included thrombocytopenia (37%), neutropenia (31%), and fatigue (27%).

Overall, AEs led to treatment discontinuation in a small percentage of patients for both monotherapy and combination regimens.

UM171 cell therapy (Zemcelpro) won conditional approval in Europe for patients with hematologic malignancies who require allogeneic hematopoietic stem cell transplantation (HSCT) after myeloablative conditioning when no other suitable type of donor cells is available.6

In accordance with this regulatory decision, the marketing of UM171 cell therapy is authorized in all 27 EU countries, as well as Iceland, Norway, and Liechtenstein. Additional filings are anticipated for UM171 in the US, Canada, United Kingdom, and Switzerland.

The efficacy and safety of UM171 was assessed in 2 independent prospective trials: a phase 1/2 study (NCT02668315) and a phase 2 study (NCT04103879) in high- and very high–risk acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndrome.7 At a median follow-up of 24 months, the estimated 2-year overall survival (OS) rate among patients treated with UM171 (n = 50) was 67% (95% CI, 54%-82%), and the progression-free survival (PFS) rate was 63% (95% CI, 50%-79%). Moreover, the 2-year cumulative incidence of nonrelapse mortality was 19% (95% CI, 8%-31%), and the incidence of relapse was 18% (95% CI, 6%-29%).

Regarding safety, the incidence of grade 2 to 4 acute graft-vs-host-disease (GVHD) was 69%; grade 3 to 4 acute GVHD occurred in 16% of patients. The rate of moderate-to-severe chronic GVHD was 7%, leading to a chronic GVHD relapse-free survival rate of 55% (95% CI, 42%-72%).

The EC approved tislelizumab (Tevimbra) in combination with platinum-containing chemotherapy as neoadjuvant therapy, followed by tislelizumab monotherapy as adjuvant therapy, for adult patients with resectable non–small cell lung cancer (NSCLC) at high risk of recurrence.8

The approval was backed by data from the phase 3 RATIONALE-315 trial (NCT04379635). The tislelizumab regimen demonstrated a statistically significant and clinically meaningful improvement in OS compared with chemotherapy plus placebo.

Previously reported data from an interim analysis also revealed a statistically significant improvement in event-free survival (EFS) for the tislelizumab arm (HR, 0.56; 95% CI, 0.40-0.79; 1-sided P = .0003).9 Furthermore, tislelizumab plus chemotherapy produced significantly higher rates of major pathological response (difference, 41.1%; 95% CI, 33.2%-49.1%; P P

Regarding safety, grade 3 or higher treatment-related AEs (TRAEs) were reported in 72.1% of patients in the tislelizumab arm versus 66.4% in the placebo arm. The most common grade 3/4 TRAEs in the experimental arm included decreased neutrophil count and decreased white blood cell count.

References

  1. European Commission grants approval of Ogsiveo (nirogacestat) for the treatment of adults with desmoid tumors. News release. August 18, 2025. Accessed August 29, 2025. https://ir.springworkstx.com/news-releases/news-release-details/european-commission-grants-approval-ogsiveor-nirogacestat
  2. FDA approves nirogacestat for desmoid tumors. FDA. Updated November 28, 2023. Accessed August 29, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nirogacestat-desmoid-tumors
  3. The European Commission has approved a tablet formulation of BeOne Medicines’ Brukinsa for use across all of its previously authorized indications. News release. BeOne Medicines. August 21, 2025. Accessed August 29, 2025. https://ir.beonemedicines.com/news/european-commission-approves-tablet-formulation-of-beone-medicines-brukinsar-for-all-approved-indications/60a73cd1-3e95-46b4-b377-578f401440b9
  4. U.S. FDA approves tablet formulation of BeOne’s Brukinsa for all approved indications. News release. BeOne. June 11, 2025. Accessed August 29, 2025. https://www.businesswire.com/news/home/20250611860939/en/U.S.-FDA-Approves-Tablet-Formulation-of-BeOnes-BRUKINSA-for-All-Approved-Indications
  5. Brukinsa. European Medicines Agency. Accessed August 29, 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/brukinsa
  6. Zemcelpro (UM171 cell therapy) receives EC authorization as the first and only cell therapy for blood cancer patients without access to suitable donor cells. News release. ExCellThera. August 27, 2025. Accessed August 29, 2025. https://www.prnewswire.com/news-releases/zemcelpro-um171-cell-therapy-receives-ec-authorization-as-the-first-and-only-cell-therapy-for-blood-cancer-patients-without-access-to-suitable-donor-cells-302539224.html
  7. Milano F, Sauvageau G, Thauvette G, et al. Infusion of UM171-expanded cord blood led to excellent survival in patients with high-risk leukemias: results from two independent phase II studies. Blood. 2023; 142(suppl 1):1040. doi:10.1182/blood-2023-187446
  8. European Commission approves Tevimbra as neoadjuvant/adjuvant NSCLC treatment ahead of late-breaking data presentation at WCLC 2025. News release. BeOne Medicines. August 27, 2025. Accessed August 29, 2025. https://ir.beonemedicines.com/news/european-commission-approves-tevimbrar-as-neoadjuvantadjuvant-nsclc-treatment-ahead-of-late-breaking-data-presentation-at-wclc/0d7553af-93d0-4616-a02b-ac223e497bca
  9. BeOne Medicines receives positive CHMP opinion for Tevimbra in neoadjuvant/adjuvant NSCLC treatment. News release. BeOne Medicines. July 28, 2025. Accessed August 29, 2025. https://ir.beonemedicines.com/news/beone-medicines-receives-positive-chmp-opinion-for-tevimbrar-in-neoadjuvantadjuvant-nsclc-treatment/a97f721c-af0c-439c-a2b5-a0e7d29eefa2