FDA Approvals, News & Updates

On December 18, 2020, the FDA approved osimertinib (Tagrisso; AstraZeneca) for adjuvant therapy after tumor resection in patients with non–small-cell lung cancer (NSCLC) and EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test. The FDA approved this application 2 months ahead of the FDA goal date and granted it a breakthrough therapy designation. Osimertinib was previously approved for the first-line treatment of metastatic NSCLC and EGFR exon 19 deletions or exon 21 L858R mutations, and for the treatment of metastatic NSCLC and EGFR T790M mutation after EGFR tyrosine kinase inhibitor therapy.

On January 14, 2021, the FDA approved crizotinib (Xalkori; Pfizer) for the treatment of young patients aged 1 to 21 years with relapsed or refractory systemic anaplastic large-cell lymphoma (ALCL) and ALK mutation. The safety and efficacy of crizotinib have not been established in older adults with this diagnosis. The FDA granted this application priority review and breakthrough therapy and orphan drug designations. Crizotinib was previously approved for patients with metastatic non–small-cell lung cancer and ALK or ROS1 mutation.

On December 16, 2020, the FDA approved margetuximab-cmkb (Margenza; MacroGenics) in combination with chemotherapy for the treatment of adults with metastatic HER2-positive breast cancer who have received ≥2 previous anti-HER2 regimens, of which at least 1 was for metastatic disease. The application for this approval received a fast-track designation.

On December 18, 2020, the FDA approved relugolix (Orgovyx; Myovant Sciences), an oral gonadotropin-releasing hormone (GnRH) receptor antagonist, for the treatment of adults with advanced prostate cancer. Relugolix is the first oral androgen-deprivation therapy (ADT) and the first oral GnRH receptor antagonist approved for patients with advanced prostate cancer. The FDA granted relugolix priority review for this indication.

On February 3, 2021, the FDA accelerated the approval of oral tepotinib (Tepmetko; EMD Serono) for adults with metastatic non–small-cell lung cancer (NSCLC) harboring MET exon 14 (METex14) skipping alterations. This approval is for treatment-naïve patients as well as for patients who have received previous therapy. Tepotinib is the second MET inhibitor approved by the FDA and should be selected for treatment based on the presence of METex14. The FDA has granted tepotinib breakthrough therapy and orphan drug designations.

On October 23, 2020, the FDA approved the FoundationOne CDx diagnostic test as a companion diagnostic to identify fusions in the NTRK1, NTRK2, and NTRK3 genes in patients who are eligible for treatment with larotrectinib (Vitrakvi).

In October and November, the FDA approved FoundationOne Liquid CDx (Foundation Medicine), a next-generation sequencing liquid biopsy test, for the identification of multiple cancers and biomarkers.

On October 2, 2020, the FDA approved the combination of nivolumab (Opdivo; Bristol Myers Squibb), a PD-1 inhibitor, and ipilimumab (Yervoy; Bristol Myers Squibb), a CTLA-4 inhibitor, for first-line treatment of adults with unresectable malignant pleural mesothelioma.

On October 14, 2020, the FDA expanded the approval of pembrolizumab (Keytruda; Merck), a PD-1 inhibitor, for the treatment of relapsed or refractory classical Hodgkin lymphoma in adults and for refractory classical Hodgkin lymphoma or classical Hodgkin lymphoma in pediatric patients that has relapsed after receiving ≥2 lines of therapy. The FDA granted pembrolizumab orphan drug and breakthrough therapy designations for this indication.

On November 13, 2020, the FDA accelerated the approval of pembrolizumab (Keytruda; Merck), a PD-1 inhibitor, plus chemotherapy, for the treatment of locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) in patients with PD-L1 (combined positive score [CPS] ≥10), as determined by an FDA-approved test.