The first innovation in stem cell mobilization for multiple myeloma in a decade, motixafortide (Aphexda™) received U.S. Food and Drug Administration approval on September 11, 2023. The drug’s clinical trial demonstrated that one dose of motixafortide plus filgrastim enabled a majority of patients to achieve the collection goal of at least six million hematopoietic stem cells.

On April 5, 2024, the U.S. Food and Drug Administration (FDA) granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu^®) for adult patients with unresectable or metastatic HER2-positive (IHC3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options. It is one of seven anticancer agents with a tumor-agnostic FDA approval.

This ONS resource was produced for educational purposes only. Refer to the full talquetamab-tgvs (Talvey™) package insert for all details. 

In two clinical trials, patients who received fruquintinib (Fruzaqla™) had an overall survival of 7.4 and 9.3 months compared to 4.8 and 6.6 months with placebo, leading to the drug’s November 2023 U.S. Food and Drug Administration approval for adults with metastatic colorectal cancer who previously received standard chemotherapy and experienced disease progression. 

In a clinical trial of 65 patients with metastatic or recurrent Merkel cell carcinoma who had not previously received systemic therapy for advanced disease, treatment with retifanlimab-dlwr (Zynyz™) achieved a 52% overall response rate and 18% complete response rate, leading the U.S. Food and Drug Administration to grant it accelerated approval in March 2023.  

After an open-label, multicenter, single-arm trial demonstrated a 56% objective response rate, with 43% achieving complete responses, and an estimated 18.4-month median duration of response, the U.S. Food and Drug Administration granted glofitamab-gxbm (Columvi™) accelerated approval in June 2023. 

On November 16, 2023, the U.S Food and Drug Administration (FDA) approved pembrolizumab (Keytruda^®) with fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma.

After a single-arm clinical trial demonstrated an overall response rate of 31.7% in 106 patients with folate receptor (FR) alpha–positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, the U.S. Food and Drug Administration granted mirvetuximab soravtansine-gynx (Elahere^®) accelerated approval on November 14, 2022.  

In August 2022, the U.S. Food and Drug Administration granted regular approval to capmatinib (Tabrecta®) for adults with metastatic non-small cell lung cancer whose tumors have a variant that leads to mesenchymal-epithelial transition exon 14 skipping. Capmatinib was granted accelerated approval for the same indication in May 2020; the regular approval in August 2022 was based on additional data from 63 patients and follow-up data establishing durability of response and clinical benefit.

On September 14, 2023, the U.S. Food and Drug Administration (FDA) approved new labeling for temozolomide (Temodar^®) under Project Renewal, an Oncology Center of Excellence (OCE) initiative that updates labeling information for older oncology drugs to ensure it is clinically meaningful and scientifically current. This is the second drug—both anticancer agents—to receive a labeling update under the pilot program. The first was for capecitabine (Xeloda^®).

In clinical trials, pirtobrutinib (Jaypirca™) was effective in restoring BTK inhibition in patients that experienced progression after previously receiving a covalent BTK inhibitor. Pirobrutinib became the first-available noncovalent (reversible) BTK inhibitor in January 2023 when the U.S. Food and Drug Administration granted it accelerated approval.

On August 14, 2023, the U.S. Food and Drug Administration approved melphalan (Hepzato™) for injection or hepatic delivery system (Hepzato Kit) containing melphalan as a liver-directed treatment for adult patients with uveal melanoma with unresectable hepatic metastases 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.

On August 14, 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to elranatamab-bcmm (Elrexfio™), a bispecific B-cell maturation antigen-directed CD3 T-cell engager, for adults 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.

On August 11, 2023, the U.S. Food and Drug Administration (FDA) approved the fixed dose combination of niraparib and abiraterone acetate (Akeega^™) plus prednisone for adult patients with deleterious or suspected deleterious BRCA variant metastatic castration-resistant prostate cancer, as determined by an FDA-approved test.

On August 9, 2023, the U.S. Food and Drug Administration granted accelerated approval to talquetamab-tgvs (Talvey^™) for adults 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.

On August 9, 2023, the U.S. Food and Drug Administration (FDA) granted regular approval to pralsetinib (Gavreto^®) for adult patients with metastatic, rearranged during transfection fusion-positive non-small cell lung cancer as detected by an FDA-approved test.

On August 2, 2023, the U.S. Food and Drug Administration (FDA) approved trifluridine and tipiracil (Lonsurf®) with bevacizumab, for metastatic colorectal cancer (mCRC) previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biologic therapy, and if RAS wild-type, an anti-EGFR therapy. FDA had previously approved single-agent trifluridine and tipiracil for the indication in September 2015.

On July 31, 2023, the U.S. Food and Drug Administration approved dostarlimab-gxly (Jemperli) with carboplatin and paclitaxel, followed by single-agent dostarlimab-gxly, for primary advanced or recurrent endometrial cancer that is mismatch repair deficient, as determined by an FDA-approved test, or microsatellite instability-high.

After clinical trials demonstrated a 43% durable objective response rate with a median 8.5-month duration, the U.S. Food and Drug Administration (FDA) granted adagrasib (Krazati™) accelerated approval in December 2022 for adult patients with locally advanced or metastatic non-small cell lung cancer with a KRAS G12C variant, as detected by an FDA-approved test, whose disease has progressed on or after at least one prior systemic therapy.

On July 20, 2023, the U.S. Food and Drug Administration (FDA) approved quizartinib (Vanflyta®) in combination with standard cytarabine and anthracycline induction and cytarabine consolidation and as maintenance monotherapy following consolidation chemotherapy for the treatment of adult patients with newly diagnosed acute myeloid leukemia that are FLT3 internal tandem duplication-positive, as detected by an FDA-approved test. FDA also approved the LeukoStrat CDx FLT3 variant assay as a companion diagnostic for quizartinib.

After clinical trial outcomes demonstrated a statistically significant difference in progression-free survival in a subgroup of patients with estrogen receptor-positive, HER2-negative, ESR1-altered disease, the U.S. Food and Drug Administration granted elacestrant’s (Orserdu™) application priority review and fast-track designation, with regular approval in January 2023.   

With an estimated duration of response rate of 90.6% at six months and 66.5% at nine months in the agent’s clinical trials, in October 2022, the U.S. Food and Drug Administration granted accelerated approval to teclistamab-cqyv (Tecvayli^®) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four previous lines of therapy.  

On June 20, 2023, the U.S. Food and Drug Administration approved talazoparib (Talzenna®) with enzalutamide for homologous recombination repair gene–variant metastatic, castration-resistant prostate cancer.

On June 15, 2023, the U.S. Food and Drug Administration granted accelerated approval to glofitamab-gxbm (Columvi™) for relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy.

The COVID-19 pandemic crushed supply chains and caused the global trading system to falter. Although many industries have rebounded, pharma is still experiencing its effects. Drug shortages directly limit healthcare providers’ ability to provide patient-centered oncology care.

On May 31, 2023, the U.S. Food and Drug Administration approved olaparib (Lynparza^®) plus abiraterone and prednisone or prednisolone for adult patients with deleterious or suspected deleterious BRCA variant, metastatic, castration-resistant prostate cancer, as determined by an FDA-approved companion diagnostic test.

In an open-label, multicenter, multicohort study that included 90 patients with relapsed or refractory follicular lymphoma who had received at least two prior lines of systemic therapy, 60% of patients receiving treatment with mosunetuzumab-axgb achieved a complete response and 80% had an overall response. On December 22, 2022, the U.S. Food and Drug Administration granted mosunetuzumab-axgb accelerated approval based on those findings.

On May 19, 2023, the U.S. Food and Drug Administration granted accelerated approval to epcoritamab-bysp (Epkinly®) for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after two or more lines of systemic therapy.

Under the U.S. Food and Drug Administration’s (FDA’s) fast-track approval process, nadofaragene firadenovec-vncg (Adstiladrin®) became the first gene therapy approved for the treatment of high-risk non-muscle invasive bladder cancer. Approximately 75% of newly diagnosed bladder cancers are classified as non-muscle invasive. Bacillus Calmette Guérin has been used as first-line therapy for more than 20 years, but eventually 30%–50% of patients stop responding, and nadofaragene firadenovec-vncg offers a new treatment option. FDA’s December 2022 approval was based on complete response (CR) and duration of response from Study CS-003, which demonstrated a 46% CR for at least one year.

On April 19, 2023, the U.S. Food and Drug Administration approved polatuzumab vedotin-piiq (Polivy®) with rituximab, cyclophosphamide, doxorubicin, and prednisone for adult patients who have previously untreated diffuse large B-cell lymphoma, not otherwise specified, or high-grade B-cell lymphoma and who have an International Prognostic Index score of 2 or greater.

On April 17, 2023, the U.S. Food and Drug Administration approved omidubicel-onlv (Omisirge^®) for use in adult and pediatric patients aged 12 years and older with hematologic malignancies receiving umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.

Once abandoned because of dose-limiting toxicities, a novel chemotherapy drug is showing even more promise as a modified prodrug. In study results published in Science Advances, researchers demonstrated how turning DRP-104 into a prodrug enables it to kill more tumor cells and activate CD8+ T cells with a “markedly improved tolerability profile.”

On April 3, 2023, the U.S. Food and Drug Administration granted accelerated approval to enfortumab vedotin-ejfv (Padcev®) with pembrolizumab (Keytruda®) for patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy.

When the U.S. Food and Drug Administration (FDA) granted pemigatinib (Pemazyre®) a new indication in August 2022, it became the first targeted therapy approved for treatment of patients with relapsed or refractory myeloid or lymphoid neoplasms (MLNs) with fibroblast growth factor receptor (FGFR) 1 rearrangement. In 2021, FDA gave pemigatinib accelerated approval for patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or other rearrangement as detected by an FDA-approved test.

On March 22, 2023, the U.S. Food and Drug Administration granted accelerated approval to retifanlimab-dlwr (Zynyz®) for adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma.

On March 16, 2023, the U.S. Food and Drug Administration approved dabrafenib (Tafinlar®) with trametinib (Mekinist®) for pediatric patients aged one year and older with low-grade glioma (LGG) that have a BRAF V600E variant who require systemic therapy. FDA also approved new oral formulations of both drugs for patients who cannot swallow pills. It is the first FDA approval of a systemic therapy for first-line treatment of pediatric patients with LGG that have a BRAF V600E variant.

On March 3, 2023, the U.S. Food and Drug Administration approved abemaciclib (Verzenio®) in combination with endocrine therapy (tamoxifen or an aromatase inhibitor) for the adjuvant treatment of adult patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative, node-positive, early breast cancer who are at high risk for recurrence.

Patients with rare cancers like intrahepatic cholangiocarcinoma seldom have many additional treatment options if their tumors become resistant to first-line therapy: From August 1, 2021–July 31, 2022, the U.S. Food and Drug Administration approved only five new agents or indications for rare tumors. On September 30, 2022, FDA added one more, giving futibatinib (Lytgobi™) accelerated approval for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 gene fusions or other rearrangements. The approval was based on the drug’s 42% overall response rate and median duration of response of 9.7 months.

On February 9, 2023, the U.S. Food and Drug Administration approved dostarlimab-gxly (Jemperli®) for adult patients with mismatch repair deficient recurrent or advanced endometrial cancer, as determined by an FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation.

On February 3, 2023, the U.S. Food and Drug Administration approved sacituzumab govitecan-hziy (Trodelvy®) for unresectable locally advanced or metastatic hormone receptor–positive, human epidermal growth factor receptor 2–negative (IHC 0, IHC 1+, or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting.

On January 27, 2023, the U.S. Food and Drug Administration approved elacestrant (Orserdu®) for postmenopausal women or adult men with estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-variant advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.

On January 26, 2023, the U.S. Food and Drug Administration approved pembrolizumab (Keytruda®) for adjuvant treatment following resection and platinum-based chemotherapy of patients with stage IB (T2a ≥ 4 cm), II, or IIIA non-small cell lung cancer.

On January 27, 2023, the U.S. Food and Drug Administration granted accelerated approval to pirtobrutinib (Jaypirca®) for relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase inhibitor.

On August 5, 2022, the U.S. Food and Drug Administration approved darolutamide (Nubeqa®) in combination with docetaxel in adults with metastatic hormone-sensitive prostate cancer. The approval was based on clinical trial findings demonstrating that the agent improved overall survival and significantly delayed time to pain progression compared to placebo.

On January 19, 2023, the U.S. Food and Drug Administration approved zanubrutinib (Brukinsa®) for the treatment of adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma.

On January 19, 2023, the U.S. Food and Drug Administration granted accelerated approval to tucatinib (Tukysa®) in combination with trastuzumab for RAS wild-type HER2-positive unresectable or metastatic colorectal cancer that has progressed following fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy.

On December 22, 2022, the U.S. Food and Drug Administration granted accelerated approval to mosunetuzumab-axgb (Lunsumio®), a bispecific, CD20-directed, CD3 T-cell engager for adult patients with relapsed or refractory follicular lymphoma who’ve received two or more lines of systemic therapy.

On December 16, 2022, the U.S. Food and Drug Administration approved nadofaragene firadenovec-vncg (Adstiladrin®) for adult patients with high-risk, Bacillus Calmette-Guérin unresponsive, carcinoma in situ, non–muscle-invasive bladder cancer with or without papillary tumors.

More than 40% of adult patients with mismatch repair deficient recurrent or advanced endometrial cancer had an overall response receiving dostarlimab-gxly (Jemperli), and more than 90% of those lasted six months or longer during the drug’s clinical trials. Those results led the U.S. Food and Drug Administration to grant the agent its original accelerated approval in 2021 for the indication. FDA expanded the accelerated approval later that year to include all recurrent or advanced solid tumors with dMMR demonstrated through an FDA-approved diagnostic test.

On December 12, 2022, the U.S. Food and Drug Administration (FDA) granted accelerated approval to adagrasib (Krazati^®), a RAS GTPase family inhibitor, for adult patients with KRAS G12C–variant locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, and who have received at least one prior systemic therapy.