10 Recent Oncology Drug Approvals

2. Pembrolizumab Approved for Adjuvant Use in Melanoma Patients. On February 15, 2019, pembrolizumab received FDA approval as an adjuvant therapy for patients with melanoma with post–complete resection lymph node involvement. Approval for use in these patients was based on the randomized EORTC1325/KEYNOTE‑054 trial (ClinicalTrials.gov identifier: NCT02362594), in which patients with completely resected stage IIIA, IIIB, or IIIC melanoma were randomized to receive pembrolizumab vs placebo. Fewer recurrences/deaths occurred in the pembrolizumab arm vs the placebo arm (26% vs 43%, respectively). The median recurrence‑free survival was 20.4 months in those receiving placebo but was not reached in the pembrolizumab arm. (Source)

3. Cabozantinib Approved for Treatment of Hepatocellular Cancer in Patients Who Previously Received Sorafenib. On January 14, 2019, cabozantinib received FDA approval for use in patients with hepatocellular carcinoma who have undergone prior treatment with sorafenib. Approval for use in these patients was based on the randomized CELESTIAL trial (ClinicalTrials.gov identifier: NCT01908426), in which patients received oral cabozantinib vs placebo. In the cabozantinib arm, the median overall survival was reported to be 10.2 months vs 8 months in the placebo arm. Median PFS was 5.2 vs 1.9 months and the overall response rate was 4.0% vs 0.4% in the cabozantinib and placebo arms, respectively. (Source)

4. Olaparib Approved as First-Line Maintenance Therapy for Advanced, BRCA-Mutated Ovarian Cancer. On December 19, 2018, olaparib received FDA approval as a maintenance therapy for those with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who harbor a deleterious or suspected deleterious derline or somatic BRCA mutation. Patients who already received platinum-based chemotherapy and experienced a complete or partial response to therapy are eligible. Approval for use in this population was based on the randomized SOLO-1 trial (ClinicalTrials.gov identifier: NCT01844986), in which patients received oral olaparib or placebo. Median PFS was 13.8 months in the placebo arm and was not reached in the olaparib arm. (Source)

5. Tagraxofusp-erzs Approved for Blastic Plasmacytoid Dendritic Cell Neoplasm. On December 21, 2018, tagraxofusp-erzs was approved for use in both adult and pediatric patients aged 2 years and older for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). Approval of the CD123-directed cytotoxin was based on the STML-401-0114 trial (ClinicalTrials.gov identifier: NCT02113982), in which patients with untreated or relapsed/refractory BPDCN received intravenous tagraxofusp-erzs under a single-arm trial. Both complete and clinical complete responses were seen in both cohort populations. Currently, the approved dose of tagraxofusp-erzs is 12 mcg/kg over a 15-minute intravenous infusion on days 1 to 5 of a 21-day treatment cycle. (Source)

6. Gilteritinib Approved for Refractory/Relapsed Acute Myeloid Leukemia (AML) With an FLT3 Mutation. On November 28, 2018, gilteritinib received FDA approval for the treatment of adult patients with relapsed or refractory AML who harbor an FLT3 mutation as detected by the LeukoStrat CDx FLT3 Mutation Assay, the companion diagnostic to detect the FLT3 mutation in these patients (this test also received FDA approval [expanded]). Approval for use in this population of patients was based on the results of the ADMIRAL trial (ClinicalTrials.gov identifier: NCT02421939), in which patients with AML and certain gene mutations received oral gilteritinib. At median follow-up, 29 of 138 patients (21%) reported complete remission (CR) or CR with partial hematologic recovery (95% CI, 14.5–28.8). Improvements in platelet or red blood cell transfusion rates were also observed. (Source)

7. Rucaparib Approved as Maintenance Therapy for Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer. On April 6, 2018, the PARP inhibitor, rucaparib, was approved for as a maintenance therapy following a complete or partial response to a platinum-based chemotherapy in those with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer. Approval for use in this population of patients is based on the results of the ARIEL3 trial in which patients were randomized to receive rucaparib or placebo. Three subgroups were analyzed: all patients, tBRCA mutations and positive homologous recombination deficiency (HRD). Median progression free survival (PFS) vs placebo was improved in all groups, especially in those with tBRCA mutataions who experienced a 16.6 mo PFS vs 5.4 months in the placebo arm. (Source)

8. Osimertinib Approved as First-Line Therapy for Patients With EGFR-Mutated Metastatic NSCLC. Patients with NSCLC whose tumors exhibit the EGFR exon 19 or exon 21 L858R mutations may now receive osimertinib as first-line therapy. On April 18, 2018, the FDA approved this therapy based on the results of the FLAURA trial (ClinicalTrials.gov identifier: NCT02296125), in which participants were randomized to receive osimertinib vs standard therapy of gefitinib or erlotinib. A subset (20%) of those randomized to undergo standard therapy received subsequent osimertinib. Estimated median PFS for the osimertinib arm was 18.9 months vs 10.2 months in the standard therapy arm; those who received osimertinib also experienced an overall response rate of 77% vs 69% with standard therapy. (Source)

9. Combination Prednisone and Abiraterone Acetate Approved for Metastatic High-Risk Castration-Sensitive Prostate Cancer (CSPC). On February 7, 2018, combination prednisone and abiraterone acetate received FDA approval for the treatment of patients with metastatic high-risk CSPC. Approval for use in this population was based on the results of the LATITUDE trial (ClinicalTrials.gov identifier: NCT01715285), in which patients received either combination treatment or placebo plus a gonadotropin-releasing hormone (GnRH) or bilateral orchiectomy. Median overall survival and time-to-initiation of chemotherapy were 34.7 months and 38.9 months, respectively, in the placebo group, whereas these endpoints were unable to be established and not reached in the treatment arm. According to the FDA, patients receiving combination therapy should also undergo treatment with a GnRH analog concurrently or undergo a bilateral orchiectomy. (Source)

10. Afatinib Indication Expands to Include Metastatic Non–Small-Cell Lung Cancer (NSCLC) With Non-Resistant Epidermal Growth Factor Receptor (EGFR) Mutations. On January 12, 2018, afatinib was granted FDA approval, broadening its first-line therapeutic indication to include patients with metastatic NSCLC who have a confirmed non-resistant EGFR mutation validated with the use of an FDA-approved test. This approval came based on the results of responses in 32 patients enrolled in either the LUX-Lung 2, LUX-Lung 3, or LUX-Lung 6 clinical trials, who received afatinib in the setting of metastatic NSCLC with certain confirmed non-resistant EGFR mutations. Afatinib-treated patients experienced a 66% overall response rate. Among the responders, 52% experienced a response duration of 12 months or longer, and 33% experienced a response duration of 18 months or longer. (Source)