FDA Drug Approvals Q3 2023

Mary L. Windle, PharmD


November 10, 2023

In This Article


Elrexfio (elranatamab)

Elranatamab is indicated for adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Elranatamab is an antibody that binds to both CD3 on T-cells and B-cell maturation antigen (BCMA), which are expressed on the surface of multiple myeloma cells. This results in cross-linking of T-cells and myeloma cells and induces a potent cytotoxic T-lymphocyte response against BCMA-expressing cells.

Accelerated approval was supported by the phase 2 MagnetisMM-3 study, which  showed the overall response rate (ORR) of 61% (35% complete response). Nat Med. 2023 Aug 15

Talvey (talquetamab)

Another bispecific antibody, talquetamab, also gained accelerated approval for adults with relapsed or refractory multiple myeloma who have received at least 4 prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. It is a bispecific humanized monoclonal antibody against CD3, a T-cell surface antigen, and GPRC5D (human G-protein coupled receptor family C group 5 member D), a tumor-associated antigen with potential antineoplastic activity.

Phase 2 findings from the MonumentTAL trial showed patients achieved an overall response rate (ORR) of 71-74%. The ORR was 64% in patients with prior T-cell redirecting therapy. J Clin Oncol. 2023;41(16; suppl):8036

Ojjaara (momelotinib)

Momelotinib is indicated for intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF (post polycythemia vera [PV] and post essential thrombocythemia [ET]), in adults with anemia. 

In the SIMPLIFY-1 multicenter, randomized, double-blind, phase III study, safety and efficacy of momelotinib was compared with ruxolitinib in 432 patients with myelofibrosis who had not received prior treatment with a JAK inhibitor. Momelotinib met the primary endpoint of noninferiority for spleen response, but not the key secondary end point of symptomatic improvement. However, more patients achieved transfusion independence with momelotinib than with ruxolitinib, and objective laboratory measures of anemia (eg, hemoglobin, serum iron, transferrin saturation) also favored momelotinib. Cancer Med. 2023 May;12(9):10612-10624

Vanflyta (quizartinib)

Quizartinib is a first-in-class tyrosine kinase inhibitor approved for newly diagnosed FLT3 internal tandem duplication–positive (ITD+) acute myeloid leukemia (AML). It was granted approval for use in combination with standard chemotherapy—cytarabine and anthracycline induction followed by cytarabine consolidation—and as maintenance monotherapy afterward. Quizartinib and its active metabolite (AC886) inhibit FLT3 kinase activity, preventing autophosphorylation of the receptor, thereby inhibiting downstream FLT3 receptor signaling and blocking FLT3-ITD–dependent cell proliferation. 

Approval was supported by the QuANTUM-First phase 3 trial. Results showed that adding quizartinib to standard chemotherapy with or without allogeneic hematopoietic stem cell transplantation, followed by continuation monotherapy for up to 3 years, resulted in improved overall survival. Median overall survival was 31.9 months for quizartinib versus 15.1 months for placebo. Lancet. 2023 May 13;401(10388):1571-1583

Aphexda (motixafortide)

Motixafortide is indicated in combination with filgrastim to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. Motixafortide is an inhibitor of C-X-C motif chemokine receptor 4 (CXCR4) and blocks binding of its cognate ligand, stromal-derived factor-1α (SDF-1α)/C-X-C motif chemokine ligand 12 (CXCL12). SDF-1α and CXCR4 play a role in trafficking and homing of human hematopoietic stem cells to the marrow compartment. 

Approval was supported by the phase 3 GENESIS trial. The primary endpoint was the proportion of patients collecting ≥6 × 106 CD34+ cells/kg within 2 apheresis procedures; the secondary endpoint was to achieve this goal in 1 apheresis. The American Society for Transplantation and Cellular Therapy (ASTCT) guidelines recommend a collection target of 3-5 × 106 CD34+ cells/kg. Motixafortide plus G-CSF (granulocyte colony-stimulating factor) enabled 92.5% to successfully meet the primary endpoint, versus 26.2% with placebo plus G-CSF (P<0.0001). Motixafortide plus G-CSF also enabled 88.8% to meet the secondary endpoint, versus 9.5% with placebo plus G-CSF (P<0.0001). Nat Med. 2023 Apr;29(4):869-879

Other hematology/oncology approvals

Lonsurf (trifluridine/tipiracil) – Indication approved for previously treated metastatic colorectal cancer in combination with bevacizumab

Hepzato (melphalan) – New kit approved for intrahepatic administration for liver metastases associated with uveal melanoma

AKeega (niraparib/abiraterone) – New fixed-dose combination for BRCA-mutated metastatic castration-resistant prostate cancer

Reblozyl (luspatercept) – Now approved as first-line treatment for anemia associated with erythropoiesis stimulating agent (ESA)–naive adults with myelodysplastic syndromes

Jemperli (dostarlimab) – Indication for endometrial cancer expanded to include combined first-line use with carboplatin and paclitaxel

Mekinist (trametinib) in combination with Tafinlar (dabrafenib) – Indication for BRAF V600E-positive solid tumors expanded to include children aged 1 year and older (previously approved for children aged 6 years and older).

Xalkori (crizotinib) – New oral pellet dosage form approved with revised dosing allows use for lower-weight patients.

Temodar (temozolomide) – Indication for anaplastic astrocytoma expanded to include adjuvant treatment for newly diagnosed tumors.

Bosulif (bosutinib) – Indication for chronic-phase (CP) Philadelphia chromosome–positive myelogenous leukemia (Ph+ CML) expanded to include children aged 1 year and older who have newly diagnosed Ph+ CML or are resistant or intolerant to prior therapy. This indication is approved for adults.


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