Pharmacist's Application to Practice
The Latest Drug Updates, Written By HOPA Members
Use these member-authored Pharmacist's Applications to Practice (PAPs) to read about the role of oncology drugs in treatment plans. Get research summaries including end points and Clinical Pearls, as well as explore the role of the hematology/oncology pharmacists in the application of these drugs.
Click here to read more Drug Updates.
Mirdametinib for adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection
(February 11, 2025) Mirdametinib is the first and only FDA approved drug for the treatment of NF1-associated PN in both adult and pediatric patient populations when surgical management is not an option.
Datopotamab deruxtecan-dlnk for unresectable or metastatic, HR-positive, HER2-negative breast cancer
(January 17, 2025) Dato-DXd is recommended in the NCCN guidelines as second-line therapy for unresectable or metastatic hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer in adults who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.
Ensartinib for ALK-positive locally advanced or metastatic non-small cell lung cancer
(December 18, 2024) The National Comprehensive Cancer Network (NCCN) lists ensartinib as a preferred first-line option for patients with NSCLC with ALK rearrangement discovered prior to or during first-line systemic therapy.
Cosibelimab-ipdl for metastatic or locally advanced cutaneous squamous cell carcinoma
(December 13, 2024) Cosibelimab-ipdl is a programmed cell death ligand1 (PD-L1) blocking antibody that is FDA-approved for the treatment of adults with metastatic cutaneous squamous cell carcinoma (mCSCC) and locally advanced cutaneous squamous cell carcinoma (laCSCC) who are not candidates for curative surgery or radiation. It is the first medication in its class to receive approval for this indication.
Zenocutuzumab-zbco for non-small cell lung cancer and pancreatic adenocarcinoma
(December 4, 2024) Zenocutuzumab-zbco is a bispecific IgG1 antibody targeting the extracellular domains of HER2 and HER3 expressed on cell surfaces, inhibiting HER2:HER3 dimerization and preventing NRG1 binding to HER3, leading to decreased cell proliferation and signaling through the phosphoinositide 3-kinase (PI3K)-AKT mammalian target of rapamycin (mTOR) pathway.
Revumenib for relapsed or refractory acute leukemia with a KMT2A translocation
(November 15, 2024) Revumenib is FDA approved for the treatment of R/R AL with a KMT2Ar translocation in adult and pediatric patients ≥ 1 years of age based on data collected from the ongoing single-arm cohort of the open-label, multicenter phase I/II AUGMENT-101 trial.
Zolbetuximab-clzb with chemotherapy for gastric or gastroesophageal junction adenocarcinoma
(October 18, 2024) Zolbetuximab-clzb is FDA-approved in combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adult patients with locally advanced unresectable or metastatic, HER2-negative gastric or gastroesophageal junction (G/GEJ) adenocarcinoma whose tumors are CLDN18.2 positive.
Inavolisib for endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative, advanced breast cancer
(October 10, 2024) Inavolisib is FDA approved as a treatment option with palbociclib and fulvestrant for adults with endocrine-resistant, PIK3CA-mutated, HR-positive, human epidermal growth-factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer. The FDA also provides guidance on approved tests to assess for alterations, including FoundationOne Liquid CDx assay.
Axatilimab-csfr for chronic graft-versus-host disease
(August 14, 2024) Axatilimab-csfr (NiktimvoTM) is a first-in-class colony stimulating factor-1 receptor (CSF-1R) blocker that binds to CSF-1 receptors on monocytes and macrophages, resulting in a decrease in the proinflammatory and profibrotic monocyte and macrophage driven effects that contribute to cGVHD. Approval was based on the AGAVE-201 trial, a phase II, randomized, open-label, multicenter study.
Vorasidenib for Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation
(August 6, 2024) Vorasidenib is approved for treatment of patients with grade 2 astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations. Vorasidenib was approved by the FDA based on the INDIGO trial, a double-blind, placebo-controlled, international, phase 3 trial, in adults with residual or recurrent grade 2 IDH-mutated gliomas.
Afamitresgene autoleucel for unresectable or metastatic synovial sarcoma
(August 2, 2024) Afami-cel is the first FDA-approved T cell receptor (TCR) gene therapy approved for adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are positive for HLA antigen(s) A*02:01P, -A*02:02P, -A*02:03P or A*02:06P, and whose tumor expresses the MAGE-A4 antigen. Afami-cel was approved under the accelerated approval pathway.
Imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia
(June 6, 2024) Efficacy was evaluated in IMerge (NCT02598661), a randomized (2:1), double-blind, placebo-controlled multicenter trial in 178 patients with MDS. Patients received an intravenous infusion of imetelstat 7.1 mg/kg or placebo in 28-day treatment cycles until disease progression or unacceptable toxicity.
Tarlatamab-dlle for extensive stage small cell lung cancer
(May 16, 2024) Efficacy was evaluated in 99 patients with relapsed/refractory ES-SCLC with disease progression following platinum-based chemotherapy enrolled in DeLLphi-301 [NCT05060016], an open-label, multicenter, multi-cohort study.
Nogapendekin alfa inbakicept-pmln for BCG-unresponsive NMIBC
(April 22, 2024) Nogapendekin alfa inbakicept is a first-in-class interleukin (IL)-15 receptor agonist and gained FDA approval in combination with BCG for adults with BCG-unresponsive NMIBC.
Tislelizumab-jsgr in the treatment of advanced or metastatic squamous cell carcinoma
(March 14, 2024) Approval for tislelizumab-jsgr for advanced or metastatic esophageal squamous cell carcinoma was based on RATIONALE-302, an open-label, randomized, active-controlled, multi-center, international, phase III clinical trial.
Eflornithine for high-risk neuroblastoma
(December 13, 2023) Eflornithine (difluoromethylornithine, DFMO, Iwilfin) is a first-in-class ornithine decarboxylase inhibitor which inhibits polyamine synthesis and restores the LIN28/Let-7 metabolic pathway. This reduces the expression of MYCN and LIN28B in MYCN-amplified neuroblastoma.
Nirogacestat (Ogsiveo) for treatment of desmoid tumors
(November 27, 2023) Nirogacestat is a first in class gamma secretase inhibitor that blocks activation of the Notch receptor. Desmoid tumors have been found to produce high amounts of the Notch protein, which promotes the growth of the tumor. Nirogacestat can specifically block the activity of gamma secretase, which is an enzyme that helps activate the Notch pathway.
Capivasertib for the treatment of metastic breast cancer
(November 16, 2023) Capivasertib (Truqap) is approved for use in combination with fulvestrant for HR-positive, HER2-negative locally advanced or metastatic breast cancer with PIK3CA/AKT1/PTEN alteration and previous progression on endocrine therapy in the metastatic setting or recurrence < 12 months of completing adjuvant therapy.
Repotrectinib for ROS1-positive locally advanced or metastatic NSCLC
(November 15, 2023) Repotrectinib is approved for the treatment of ROS1-positive locally advanced or metastatic NSCLC for patients who have previously received ROS1 tyrosine kinase inhibitors (TKIs) or are TKI-naïve. Approval was based on data collected in the ongoing single arm, global multicenter, open-label phase 2 TRIDENT-1 trial.1
Fruquintinib for refractory metastatic colorectal cancer
(November 8, 2023) The FDA approved fruquintinib for adult patients with metastatic colorectal cancer (mCRC) who received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate, an anti-EGFR therapy.
Momelotinib for the treatment of myelofibrosis
(September 15, 2023) Momelotinib works by inhibiting disease drivers such as JAK1 and JAK2, which contribute to the pathogenesis of myelofibrosis (MF). Unlike other JAK inhibitors, momelotinib also inhibits activin A receptor type 1 (ACVR1), which reduces liver hepcidin expression and increases iron availability for erythropoiesis.
Hepzato Kit™ for uveal melanoma with unresectable hepatic metastasis
(August 14, 2023 ) Hepzato Kit™ (melphalan) for Injection/Hepatic Delivery System (HDS) is FDA approved as a liver-directed therapy for treatment of adult patients with uveal melanoma with unresectable hepatic metastasis affecting less than 50% of the liver and no extrahepatic disease, or extrahepatic disease limited to the bone, lymph nodes, subcutaneous tissues, or lung that is amenable to resection or radiation.
Elranatamab-bcmm for multiple myeloma
(August 14, 2023) The FDA granted accelerated approval to elranatamab-bcmm (Elrexfio, Pfizer, Inc.), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager.
Niraparib and Abiraterone Acetate (Akeega™)
(August 11, 2023) The FDA approved the fixed dose combination of niraparib and abiraterone acetate with prednisone, for adult patients with deleterious or suspected deleterious BRCA-mutated castration-resistant prostate cancer (mCRPC), as determined by an FDA-approved test.
Talquetamab-tgvs for the treatment of adult patients with relapsed or refractory multiple myeloma
(August 9, 2023) FDA approves Talvey (talquetamab-tgvs) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody.
Quizartinib for newly diagnosed acute myeloid leukemia
(July 20, 2023) The FDA approved quizartinib (Vanflyta, Daiichi Sankyo, Inc.) with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy.
Glofitamab-gxbm for selected relapsed or refractory large B-cell lymphomas
(June 15, 2023) The FDA granted accelerated approval to glofitamab-gxbm (Columvi, Genentech, Inc.) for relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified (DLBCL, NOS) or large B-cell lymphoma (LBCL) arising from follicular lymphoma.
Epcoritamab-bysp for relapsed or refractory diffuse large B-cell Lymphoma
(May 19, 2023) FDA grants accelerated approval to epcoritamab-bysp for relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma.
Retifanlimab-dlwr for MCC
(March 22, 2023) FDA grants accelerated approval to retifanlimab-dlwr for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC).
Sacituzumab govitecan-hziy for HR-positive breast cancer
(February 3, 2023) Sacituzumab govitecan-hziy is recommended in the National Comprehensive Cancer Network (NCCN) guidelines as second-line therapy for recurrent unresectable or metastatic, HR-positive/HER2-negative or triple-negative breast cancer.
Elacestrant for (HR) Positive, (HER2) Negative metastatic breast cancer
(January 27, 2023) Elacestrant for Hormone Receptor (HR) Positive, Human Epidermal Growth Factor Receptor 2 (HER2) negative metastatic breast cancer.
Pirtobrutinib for relapsed or refractory MCL
(January 27, 2023) Learn about the potential role for pirtobrutinib in the treatment of relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor.
Mosunetuzumab-axgb for relapsed or refractory follicular lymphoma
(December 22, 2022) FDA grants accelerated approval to mosunetuzumab-axgb for relapsed or refractory follicular lymphoma.
Adstiladrin (nadofaragene firadenovec-vncg) for high-risk, non-muscle-invasive bladder cancer
(December 16, 2022) FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer.
Adagrasib for KRAS G12C-mutated NSCLC
(December 12, 2022) FDA grants accelerated approval to adagrasib for adult patients with KRAS G12C¬-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.
Olutasidenib for relapsed or refractory AML
(December 1, 2022) FDA approves olutasidenib (Rezlidhia) capsules for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test.
Mirvetuximab soravtansine-gynx for FRα positive, platinum-resistant epithelial ovarian, fallopian tube, or peritoneal cancer
(November 14, 2022) FDA grants accelerated approval to mirvetuximab soravtansine-gynx for FRα positive, platinum-resistant epithelial ovarian, fallopian tube, or peritoneal cancer.
Teclistamab-cqyv for relapsed or refractory multiple myeloma
(October 25, 2022) FDA approves teclistamab-cqyv for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Tremelimumab for uHCC and mNSCLC
(October 21, 2022) Read our Pharmacist's Application to Practice about tremelimumab for unresectable hepatocellular carcinoma (HCC) and metastatic non-small cell lung cancer (NSCLC).
Futibatinib for cholangiocarcinoma
(September 30, 2022) FDA grants accelerated approval to futibatinib (Lytgobi, Taiho Oncology, Inc.) for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.