FDA and EMA Oncology Drug Approvals in Q3 2025 Included Treatments for 3 Rare Brain Tumors

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved 21 new or expanded indications for previously approved agents and 6 new oncology agents in the third quarter of 2025. The approvals included approximately 25 solid tumor types and 6 hematologic malignancies. Approximately half of the approvals were for biologics or biosimilars; there were 7 biosimilar and 8 biologic approvals, only 1 of which was a new agent. One biosimilar was approved by both the FDA and the EMA (denosumab-nxxp, Bilprevda^®, Organon and Henlius/Shanghai Henlius Biotech), and 2 additional agents were approved by just the FDA (denosumab-kyqq, Aukelso™, Biocon Biologics Ltd; denosumab-qbde, Xtrenbo™, Gedeon Richter and Hikma Pharmaceuticals) for treatment of skeletal-related events in advanced malignancies and giant cell tumor of bone.

Additional highlights from the third quarter of 2025

• Drug approvals encompassed 15 different modes of action; the most common were PD-1 inhibitors (6 approvals), RANKL inhibitors (7 approvals), EGFR inhibitors (2 approvals), MEK inhibitors (2 approvals), and bispecifics (2 approvals)
• The tumor types with the most approvals were bone (7), non-small cell lung cancer (NSCLC; 6), brain (3), breast cancer (3), and head and neck squamous cell carcinoma (3)
• Pembrolizumab and denosumab biosimilars accounted for 44% of Q3 approvals by both agencies
• Treatments for 3 rare brain tumors were approved
  • Under accelerated approval, the FDA approved dordaviprone (Modeyso™; Jazz Pharmaceuticals), the first FDA approval of a systemic therapy for H3 K27M-mutant diffuse midline glioma, an ultra-rare and aggressive brain tumor that primarily affects children and young adults
  • The EMA approved vorasidenib (Voranigo^®; Les Laboratoires Servier) for treatment of astrocytoma or oligodendroglioma, 2 rare, malignant brain tumors
• The FDA also approved a new gemcitabine intravesical system (Inlexzo™; Janssen Biotech) for treatment of bladder cancer, the first intravesical system enabling extended release of gemcitabine into the bladder
• A new, subcutaneously administered formulation of pembrolizumab (Keytruda Qlex™ (pembrolizumab and berahyaluronidase alfa-pmph; Merck) was approved by the EMA and by the FDA for several tumor types, including tumor mutational burden-high solid tumors
• There were 7 denosumab biosimilar approvals, including 3 by the FDA and 4 by the EMA
• 2 products that previously received accelerated approval gained full approval by the FDA
  • Lazertinib mesylate (Lazcluze^®; Janssen Biotech) for the treatment of NSCLC
  • Pembrolizumab (Keytruda^®; Merck), for treatment of Hodgkin lymphoma and primary mediastinal large B-cell lymphoma
• The FDA also approved 3 companion diagnostics
  • The Guardant360^® CDx assay to identify patients with breast cancer with ESR1 mutations who may be eligible for treatment with imlunestrant (Inluriyo™; Eli Lilly)
  • The Oncomine™ Dx Express Test (Life Technologies Corporation), to aid in detection of EGFR exon 20 insertion mutations in patients with NSCLC who may be eligible for treatment with sunvozertinib (Zegfrovy^®; Dizal Jiangsu)
  • The Oncomine Dx Target Test (Life Technologies Corporation), to aid in detecting HER2 (ERBB2) tyrosine kinase domain-activating mutations in patients with nonsquamous NSCLC who may be eligible for treatment with zongertinib (Hernexeos^®; Boehringer Ingelheim)

The tables below summarize key oncology approvals in Q3 2025. Treatments in bold font represent new agent approvals; all others are biosimilars, indication expansions, or conversion from accelerated to full approval (FDA).

FDA and EMA Approvals

^aPlease see each product’s prescribing information and summary of product characteristics for full indications and labeling information.

^bBiosimilar.

^cAccelerated approval.

^dBiologic.

^eConditional marketing authorization.

ALL, acute lymphocytic leukemia; BCMA, B-cell maturation antigen; CD3, cluster of differentiation 3; CML, chronic myeloid leukemia; CSF1R, colony stimulating factor 1 receptor; dMMR, mismatch repair deficient; EGFR, epidermal growth factor receptor; EMA, European Medicines Agency; ER, estrogen receptor; FDA, US Food and Drug Administration; GEJ, gastroesophageal junction; GnRH, gonadotropin releasing hormone; HCC, hepatocellular carcinoma; HER2, human epidermal growth factor receptor 2; HNSCC, head and neck squamous cell carcinoma; IDH, isocitrate dehydrogenase; MEK, mitogen-activated protein kinase kinase; MOA, mechanism of action; MPM, malignant pleural mesothelioma; MSI-H, microsatellite instability-high; NSCLC, non-small cell lung cancer; PD-1, programmed cell death protein 1; Ph+, Philadelphia chromosome-positive; RANKL, receptor activator of nuclear factor kappa beta; RCC, renal cell carcinoma; SC, subcutaneous; SCC, squamous cell carcinoma; TBM-H, tumor mutational burden-high.

Sources:

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=reportsSearch.process

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-21-24-july-2025

https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-15-18-september-2025

https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications