On October 24, 2019, a marketing authorization valid through the European Union (EU) was issued for gilteritinib monotherapy for adult patients who have relapsed or refractory acute myeloid leukemia (AML) with an Fms‐like tyrosine kinase 3 (FLT3) mutation. Gilteritinib inhibits FLT3 receptor signaling and proliferation in cells exogenously expressing FLT3 including FLT3 internal tandem duplication (ITD), FLT3 D835Y, and FLT3 ITD D835Y, and it induced apoptosis in leukemic cells expressing FLT3 ITD. The recommended starting dose of gilteritinib is 120 mg (three 40 mg tablets) once daily. Gilteritinib was evaluated in one, phase III, open‐label, multicenter, randomized study of gilteritinib (n = 247, gilteritinib arm) versus salvage chemotherapy (n = 124, salvage chemotherapy arm) in patients with relapsed or refractory AML with FLT3 mutation. Overall survival (OS) was statistically significantly different between the two groups with a median OS of 9.3 months in the gilteritinib arm compared with 5.6 months for salvage chemotherapy (hazard ratio, 0.637; 95% confidence interval, 0.490–0.830; p = .0004 one‐sided log‐rank test). The most common adverse reactions with gilteritinib treatment were blood creatine phosphokinase increase, alanine aminotransferase increase, aspartate aminotransferase increase, blood alkaline phosphatase increase, diarrhea, fatigue, nausea, constipation, cough, peripheral edema, dyspnea, dizziness, hypotension, pain in extremity, asthenia, arthralgia, and myalgia. The objective of this article is to summarize the scientific review of the application leading to regulatory approval in the EU. Implications for Practice Xospata was approved in the European Union as monotherapy for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an Fms‐like tyrosine kinase 3 (FLT3) mutation. Gilteritinib resulted in a clinically meaningful and statistically significant improvement of overall survival compared with salvage chemotherapy. At the time of the marketing authorization of gilteritinib, there were no approved standard therapies specifically for adult patients diagnosed with relapsed or refractory AML with FLT3 mutation. In terms of safety, the overall accepted safety profile was considered manageable.

Nivolumab was registered in Russia in December 2016 as a monotherapy for advanced renal cell carcinoma (RCC) and it remains a second‑line treatment choice for patients with disease progression after the use of tyrosine kinase inhibitors. Even though immunotherapy has already proven to be an effective approach for the treatment of RCC, predictive biomarkers for the rational selection of patients remain unidentified.Seventy‑five patients with metastatic renal cell carcinoma (mRCC) who received nivolumab in the 2nd and subsequent lines of therapy from 2015 to 2020 under the expanded access program were enrolled in this study. The objective response rate was 21,3 %. Median progression‑free survival (PFS) was 5,5 months. Median overall survival (OS) was not reached.To analyze molecular biomarkers correlated with the response to immunotherapeutic treatment, we performed whole‑transcriptome RNA sequencing of 16 samples (FFPE) in 15 patients with the assessment of the expression level for individual genes (PDCD1, CD274, CD8A, CD8B, CD4) and gene signatures (Angio, Teff, Myeloid Inflammation).Disease control rates were not different for the subgroups of patients with high and low expression of any of the signatures examined, and further principal component analysis did not reveal clustering of patients with and without objective response.Further studies on a larger sample of patients will help confirm or deny the predictive role of biomarkers selected for analysis in a heterogeneous population of RCC patients.