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FDA Grants Orphan Drug Designation to MB-106 for Waldenström Macroglobulinemia

Talquetamab Monotherapy Shows Early Promise in Relapsed/Refractory Multiple Myeloma
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The European Medicines Agency has verified its type II variation application to extend the indication for lisocabtagene maraleucel to include the treatment of adult patients with diffuse large B-cell lymphoma, high grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and grade 3B follicular lymphoma, who are refractory or have relapsed within 12 months of initial therapy and are candidates for hematopoietic stem cell transplant.

Dr Daniel Pollyea comments on biomarker testing in acute myeloid leukemia and the potential role for RARA testing.

A review of treatment options for patients with newly diagnosed acute myeloid leukemia and differences between patients who are fit vs unfit for chemotherapy.

Richard Stone, MD, and Eunice Wang, MD, explain how molecular biomarker testing influences their treatment decision-making in patients with acute myeloid leukemia (AML).

Drs Richard Stone and Eunice Wang review the clinical profile of a 67-year-old man with newly diagnosed acute myeloid leukemia (AML) and an IDH2 mutation.

The addition of venetoclax to ibrutinib resulted in a high rate of minimal residual disease negativity in the blood and marrow of patients with previously untreated chronic lymphocytic leukemia.

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted unanimously in favor of the benefit of betibeglogene autotemcel for the treatment of transfusion-dependent β-thalassemia in adult and pediatric patients with a non–β0/β0 genotype.

RG6234, a novel T-cell engaging bispecific antibody with a 2:1 configuration, induced high response rates and early evidence of durability for patients with relapsed/refractory multiple myeloma.

Heinz Gisslinger, MD, discusses the 6-year event-free survival data from the phase 3 PROUD-PV and CONTI-PV trials.

Nicholas J. Short, MD, discusses updated results from a single-arm phase 2 study investigating ponatinib plus blinatumomab in patients with Philadelphia chromosome–positive acute lymphoblastic leukemia.

The bispecific antibody REGN5458 elicited rapid responses that were further characterized by their depth, durability, and low incidence of cytokine release syndrome in patients with relapsed/refractory multiple myeloma.

Treatment with ropeginterferon alfa-2b-njft induced low symptom burden and low phlebotomy requirement compared with best available treatment in patients with polycythemia vera, according to long-term data from the phase 3 PROUD-PV and CONTINUATION-PV trials.

Bezuclastinib showcased early signs of clinical activity in that it resulted in a meaningful reduction in serum tryptase levels, as well as reductions in mast cell burden and KIT D816V variant allele frequency, in adult patients with advanced systemic mastocytosis.

The combination of talquetamab and daratumumab led to early onset and durable responses that deepened over time in patients with heavily pretreated multiple myeloma, most of whom were anti-CD38 refractory, according to findings from the phase 1b TRIMM-2 study.

The combination of epcoritamab and R-CHOP produced high overall response rates and complete metabolic response rates with a manageable toxicity profile in patients with diffuse large B-cell lymphoma.

The phase 3 BRUIN CLL-313 trial is currently recruiting patients with treatment naïve chronic lymphocytic leukemia or small lymphocytic lymphoma to evaluate the efficacy and safety of pirtobrutinib monotherapy vs bendamustine plus rituximab.

Zandelisib, a potent and selective oral PI3Kδ inhibitor, elicited a high overall response rate as a single agent in heavily pretreated patients with relapsed or refractory follicular lymphoma, according to data from the phase 2 TIDAL trial.

The combination of obinutuzumab plus chemotherapy delivered superior long-term progression-free survival benefit for patients with treatment-naïve follicular lymphoma.

Treatment with the R-CODOX-M and R-IVAC regimens elicited similar efficacy to dose-adjusted infused DA-EPOCH-R in patients with newly diagnosed, high-risk Burkitt lymphoma.

The longest duration of reduction in red blood cell transfusion dependence was reported among patients with β-thalassemia who received continued treatment with luspatercept-aamt in the updated data from the phase 3 BELIEVE trial.

The triplet combination regimen comprised of quizartinib, decitabine, and venetoclax elicited encouraging responses in heavily pretreated patients with relapsed/refractory FLT3-ITD–mutated acute myeloid leukemia who were previously exposed to a FLT3 inhibitor.

The first-in-class, subcutaneously administered T-cell–engaging bispecific antibody epcoritamab demonstrated deep and durable responses in patients with relapsed/refractory large B-cell lymphoma.

Musa Yilmaz, MD, discusses composite complete remission and bone marrow transplant rates observed in a phase 1/2 trial in patients with FLT3-ITD–mutated acute myeloid leukemia.

Decitabine demonstrated a comparable overall survival and rate of hematopoietic stem cell transplant in addition to a lower incidence of adverse effects vs induction chemotherapy with daunorubicin and cytarabine in older patients at least 60 years of age with acute myeloid leukemia.

Time-limited targeted therapy with venetoclax plus obinutuzumab with or without ibrutinib demonstrated superior progression-free survival outcomes compared with standard chemoimmunotherapy in patients with chronic lymphocytic leukemia.









































