FDA Approvals, News & Updates

The past week in oncology-related news includes shortages of crucial pediatric cancer drug, results of a study of racial disparities in multiple myeloma, and new drug on the horizon for HER2 metastatic breast cancer.

FDA issues finalized guidelines for use of in vitro diagnostics in oncology drug trials and warnings on vaping; new findings report patients bear more costs when MBC treatment is not concordant with NCCN guidelines.

On September 26, 2019, the FDA approved daratumumab (Darzalex; Janssen Biotech), in combination with bortezomib (Velcade), thalidomide (Thalomid), and dexamethasone, for first-line treatment of multiple myeloma in patients eligible for autologous stem-cell transplant (ASCT). In June 2019, the FDA approved the combination of daratumumab plus lenalidomide (Revlimid) and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for ASCT. The FDA granted this application a priority review.

On September 17, 2019, the FDA accelerated the approval of a new combination of pembrolizumab (Keytruda; Merck) plus lenvatinib (Lenvima; Eisai) for patients with advanced endometrial carcinoma that is not associated with microsatellite instability high (MSI-H) or mismatch repair–deficient (dMMR) tumors in patients whose disease progressed after systemic therapy and who are candidates for curative surgery or radiation. The FDA granted this application priority review and a breakthrough therapy designation.

On September 17, 2019, the FDA approved a new indication for apalu­tamide (Erleada; Janssen Biotech) for the treatment of patients with metastatic castration-sensitive prostate cancer (CSPC). Apalutamide was initially approved in 2018 for the treatment of nonmetastatic castration-resistant prostate cancer. The FDA granted apalutamide priority review for the new indication.

On August 2, 2019, the FDA approved pexidartinib (Turalio; Daiichi Sankyo) capsules, a kinase inhibitor, for adults with symptomatic tenosynovial giant-cell tumor (TGCT), which is associated with severe morbidity or functional limitations, who are not candidates for surgery. Pexidartinib is the first systemic therapy approved for patients with TGCT. The FDA granted pexidartinib a priority review and breakthrough therapy and orphan drug designations.

On August 16, 2019, the FDA approved fedratinib (Inrebic; Celgene/Impact Biomedicines), an oral kinase inhibitor, for adults with intermediate-2 or high-risk primary or secondary—post–polycythemia vera (PV) or post–essential thrombocythemia (ET)—myelofibrosis, a rare bone marrow disorder. The FDA granted fedratinib priority review and an orphan drug designation.

On August 15, 2019, the FDA accelerated the approval of entrectinib (Rozlytrek; Genentech), an oral kinase inhibitor, for the treatment of adults and adolescents whose cancer involves neurotrophic tyrosine receptor kinase (NTRK) gene fusion that has no effective treatments. The FDA granted entrectinib a priority review and breakthrough therapy and orphan drug designations. This is the third cancer drug approved by the FDA based on a common biomarker and the second drug for patients with NTRK gene fusion.

On July 30, 2019, the FDA approved darolutamide (Nubeqa; Bayer HealthCare), an oral androgen receptor inhibitor, for the treatment of patients with nonmetastatic castration-resistant prostate cancer (CRPC). This approval was based on a multicenter, double-blind, placebo-controlled clinical trial known as ARAMIS. The FDA granted this application priority review.

On September 26, 2019, the FDA approved daratumumab (Darzalex; Janssen) in combination with bortezomib, thalidomide, and dexamethasone for the treatment of newly diagnosed patients with multiple myeloma who are eligible for autologous stem-cell transplant (ASCT). The FDA granted this application priority review.