February 2021, Vol 12, No 1

Cellular immunotherapy was featured prominently during ASH 2020, with many presentations detailing deep and durable responses in a range of hematologic malignancies, mostly large B-cell lymphoma.

In a phase 3 clinical trial, ruxolitinib (Jakafi) led to superior overall response rates and durable responses compared with best available therapy (ie, control group) in patients with steroid-dependent or steroid-refractory chronic graft-versus-host disease (GVHD), with acceptable safety, according to results presented at ASH 2020.

Asciminib, an investigational first-in-class STAMP (specifically targeting the ABL myristoyl pocket) inhibitor, was superior to standard treatment with bosutinib (Bosulif) in patients with chronic-phase chronic myeloid leukemia (CML) who previously received 2 or more tyrosine kinase inhibitors (TKIs) in the phase 3 ASCEMBL clinical trial. The results were presented at a late-breaker session at ASH 2020 by lead investigator Andreas Hochhaus, MD, Director, Department of Hematology and Oncology, Klinik für Innere Medizin II, Jena, Germany.

Single-agent mosunetuzumab is a safe and clinically promising chemotherapy-free therapy for elderly and unfit patients with untreated diffuse large B-cell lymphoma (DLBCL), said Adam J. Olszewski, MD, Associate Professor of Medicine, Brown University, Providence, RI, at ASH 2020. Dr Olszewski reported the results of a phase 1/2 clinical study of mosunetuzumab in patients with DLBCL.

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 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 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 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 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 December 18, 2020, the FDA approved selinexor (Xpovio; Karyopharm Therapeutics) in combination with bortezomib (Velcade) and dexamethasone for the treatment of adults with multiple myeloma after ≥1 previous therapies. The FDA granted this indication an orphan drug designation.