February 2020, Vol 11, No 1 | Payers’ Perspectives In Oncology | Including ASH 2019 Highlights

Orlando, FL—The investigational B-cell maturation antigen (BCMA)-­directed chimeric antigen receptor (CAR) T-cell therapy known as JNJ-4528 induced responses in 100% of 29 evaluable patients with heavily pretreated relapsed or refractory multiple myeloma, according to the results of the phase 1b/2 CARTITUDE-1 clinical trial reported at ASH 2019.

Orlando, FL—An investigational oral form of azacitidine (CC-486) as maintenance therapy induced a statistically significant improvement in overall survival (OS) compared with placebo in patients with newly diagnosed acute myeloid leukemia (AML) who achieved a complete response or complete response with incomplete hematologic recovery after treatment with induction chemotherapy.

Orlando, FL—Almost 50% of patients with chronic lymphocytic leukemia (CLL) who received treatment with the triplet of acalabrutinib (Calquence), venetoclax (Venclexta), and obinu­tuzumab (Gazyva) as first-line therapy achieved undetectable minimal residual disease (MRD) in the bone marrow after only 8 monthly cycles of therapy, according to data presented at ASH 2019.

Orlando, FL—Zanubrutinib (Brukinsa), a novel Bruton tyrosine kinase (BTK) inhibitor—which was approved by the FDA in November 2019 for the treatment of mantle-cell lymphoma—achieved high overall response rate (ORR) and durable responses in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), including those with high-risk cytogenetics, according to findings presented at ASH 2019.

Orlando, FL—A new CD45-targeting antibody radiation-conjugate, iodine-131 (I-131) apamistamab, may be a less toxic alternative to today’s standard practice of chemotherapy-based lymphodepletion regimens before initiation of adoptive cell therapy, according to results presented at ASH 2019.

On December 16, 2019, the FDA approved a new indication for enzalutamide (Xtandi; Astellas Pharma) for the treatment of patients with metastatic castration-sensitive prostate cancer (CSPC). Enzalutamide was previously approved for patients with castration-­resistant prostate cancer.

On December 27, 2019, the FDA accelerated the approval of a new indication for olaparib (Lynparza; AstraZeneca) for the maintenance treatment of adults with metastatic pancreatic adenocarcinoma associated with a deleterious or suspected deleterious germline BRCA mutation, as detected by an FDA-approved test, and whose disease did not progress during ≥16 weeks of a first-line platinum-based chemotherapy regimen. Olaparib has been previously approved for ovarian cancer and for breast cancer associated with BRAF mutation.

On January 8, 2020, the FDA accelerated the approval of a new indi­cation for pembrolizumab (Keytruda; Merck & Co) for the treatment of Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk, non–muscle invasive bladder cancer and carcinoma in situ, with or without papillary tumors, in patients who are ineligible for or who have elected not to undergo cystectomy. Pembrolizumab has been previously approved for many indications.

On December 23, 2019, the FDA accelerated the approval of fam-trastuzumab deruxtecan-nxki (Enhertu; Daiichi Sankyo), a HER2-directed antibody and topoisomerase inhibitor conjugate, for the treatment of adults with unresectable or metastatic HER2-positive breast cancer after ≥2 previous anti-HER2–based regimens in the metastatic setting. The FDA granted fam-trastuzumab deruxtecan-nxki a breakthrough therapy designation.

On January 9, 2020, the FDA accelerated the approval of avapritinib (Ayvakit; Blueprint Medicines Corporation), a kinase inhibitor, for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. This approval includes GIST that harbors a PDGFRA D842V mutation, which is the most common exon 18 mutation. The FDA granted breakthrough therapy and orphan drug designations to avapritinib.