Olutasidenib was approved for the treatment of AML after it was shown in a phase I/II clinical trial that 32 percent of patients treated with the molecularly targeted therapeutic had complete remission, meaning that there was no evidence of disease and full recovery of blood counts (407). Not only does the approval of olutasidenib increase treatment options for patients with IDH1-mutated AML, but there is also preliminary evidence that patients may respond longer to olutasidenib compared to the other IDHI-targeted therapy (407). A potential side effect observed among patients treated with olutasidenib is differentiation syndrome. The condition is caused by a large, rapid release of immune molecules called cytokines from leukemia cells and can lead to fever, cough, troubled breathing, build-up of excess fluid around the heart and lungs, low blood pressure, and kidney failure, but is generally readily treated with full resolution. The FDA approval is accompanied by a warning highlighting the risk of this potentially fatal adverse effect. In July 2023, the FDA approved a second new molecularly targeted therapeutic, quizartinib (Vanflyta), for the treatment of AML. Quizartinib was approved for treating adults who have newly diagnosed AML that tests positive for a mutated FLT3 gene known as FLT3 internal tandem duplication (ITD). Mutations in the FLT3 gene promote the multiplication and survival of AML cells in 25 to 30 percent of cases, and patients with this type of AML have particularly poor outcomes (408). The approval was based on results from a phase III clinical trial showing that patients who received quizartinib had a 22 percent reduced risk Recent Advances Against Blood Cancers In the 12 months from August 1, 2022, to July 31, 2023, the U.S. Food and Drug Administration made numerous decisions that are transforming the lives of patients with a wide array of hematologic cancers, including the following: 2022 AUGUST Myeloid/Lymphoid Neoplasm Pemigatinib (Pemazyre), a molecularly targeted therapeutic, is approved. OCTOBER Multiple Myeloma Teclistamab-cqyv (Tecvayli), a T-cell engaging bispecific antibody (a type of immunotherapeutic), is approved. NOVEMBER Hodgkin Lymphoma Brentuximab vedotin (Adcetris) is a molecularly targeted therapeutic approved in combination with chemotherapeutics for patients 2 years of age and older. This is the first pediatric approval for this therapeutic. DECEMBER Acute Myeloid Leukemia Olutasidenib (Rezlidhia), a molecularly targeted therapeutic, is approved. Follicular Lymphoma Mosunetuzumab-axgb (Lunsumio), a T-cell engaging bispecific antibody (a type of immunotherapeutic), is approved. 2023 JANUARY Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma Zanubrutinib (Brukinsa), a molecularly targeted therapeutic, is approved. Mantle Cell Lymphoma Pirtobrutinib (Jaypirca), a molecularly targeted therapeutic, is approved. APRIL Stem Cell Transplant Omidubicel-onlv (Omisirge) is a cell-based therapy approved for use in adult and pediatric patients (12 years and older) with hematologic cancers who are planned for umbilical cord blood transplantation to reduce the time to neutrophil recovery and the incidence of infection. MAY-JUNE Diffuse Large B-cell Lymphoma Epcoritamab-bysp (Epkinly), a T-cell engaging bispecific antibody (a type of immunotherapeutic), is approved in May 2023. Glofitamab-gxbm (Columvi), a T-cell engaging bispecific antibody (a type of immunotherapeutic), is approved in June 2023. JULY Acute Myeloid Leukemia Quizartinib (Vanflyta), a molecularly targeted therapeutic, is approved. SIDEBAR 37 AACR Cancer Progress Report 2023 Advancing the Frontiers of Cancer Science and Medicine 94
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