fda-approvals-june-2019

New Cancer Treatments Approved in July

  • Selinexor, a New Treatment for Multiple Myeloma. The United States Food and Drug Administration (FDA) granted accelerated approval to selinexor (Xpovio®, Karyopharm Therapeutics) in combination with dexamethasone for patients with relapsed/refractory multiple myeloma who have received at least 4 prior therapies, including 2 proteasome inhibitors, 2 immunomodulatory agents, and a CD38-targeted monoclonal antibody. This approval was based on results in a subgroup of 83 heavily refractory patients on the phase IIb STORM trial, in which the overall response rate (ORR) to selinexor plux low-dose dexamethasone was 25.3%, with a median duration of response of 3.8 months. Confirmation of these results in the ongoing phase III BOSTON trial will be required for selinexor to achieve full approval.

 

  • Darolutamide Gets New Indication in Prostate Cancer. Darolutamide (Nubeqa®, Bayer), an androgen receptor inhibitor, was approved for treatment of patients with nonmetastatic castration-resistant prostate cancer (nmCRPC). The phase III ARAMIS trial compared efficacy of darolutamide plus androgen deprivation therapy (ADT) to placebo plus ADT in 1,509 patients with nmCRPC with a prostate specific antigen (PSA) doubling time of less than 10 months and a PSA level of at least 2 ng/mL. Darolutamide significantly improved the median metastasis-free survival (MFS), from 18.4 months with placebo plus ADT to 40.4 months for darolutamide plus ADT (P < .0001). Darolutamide is the third antiandrogen to be approved for this indication.

 

  • Pembrolizumab Approved for Esophageal Cancer. The PD-1 inhibitor pembrolizumab (Keytruda®, Merck) was approved for use as a monotherapy in patients with advanced squamous cell carcinoma of the esophagus with a PD-L1 combined positive score ≥ 10 with disease progression after one or more prior lines of systemic therapy. This is the first immunotherapy to be approved for this indication. The approval was based on results from the KEYNOTE-181 trial, in which pembrolizumab monotherapy resulted in a median overall survival (OS) of 9.3 months compared to 6.7 months with chemotherapy (HR 0.69, P = .0074) in patients with advanced esophageal cancer with CPS ≥ 10.

 

  • EMA Greenlights Histology Independent NTRK Inhibitor. The European Medicines Agency (EMA) gave a positive opinion recommending the approval of larotrectinib (Vitrakvi®, Bayer), a neurotrophic tyrosine receptor kinase (NTRK) inhibitor, for the treatment of any solid tumor harboring an NTRK gene fusion. Approved in the United States in November 2018, larotrectinib is the first ‘histology independent’ tumor treatment. This decision was based on results from two phaseI/II studies that evaluated larotrectinib in patients with NTRK-positive solid tumors. In these studies, larotrectinib was associated with an ORR of 75%, with 71% of responding patients remaining in response after 1 year.

 

  • Positive Opinion for Atezolizumab in TNBC. The EMA gave a positive opinion endorsing atezolizumab (Tecentriq®, Roche) in combination with nab-paclitaxel for the treatment of patients with advanced triple-negative breast cancer (TNBC) with a PD-L1 expression ≥ 1%. Atezolizumab was approved for this indication in the United States in March 2019. This recommendation from the EMA is based on results from the phase III IMpassion130 trial, in which the addition of atezolizumab to nab-paclitaxel resulted in a 38% reduction in the risk of progression or death compared to nab-paclitaxel alone in PD-L1-positive patients with advanced, unresectable TNBC.

FDA-EMA-July-2019