The FDA has approved use of dinutuximab (Unituxin [yoo ni tux' in] – United Therapeutics) in combination with interleukin-2 (IL-2), granulocyte-macrophage colony-stimulating factor (GM-CSF), and isotretinoin for treatment of children with high-risk neuroblastoma who previously responded to first-line therapies. Dinutuximab is a monoclonal antibody that binds to GD2, a glycolipid that is overexpressed on the surface of neuroblastoma cells.1

Dinutuximab received a priority review and orphan drug designation. Approval was based on the results of an open-label trial in 226 patients with high-risk neuroblastoma that had at least a partial response to induction chemotherapy, autologous stem cell transplantation, and radiation. Patients were randomized to receive a combination of dinutuximab, GM-CSF, IL-2, and isotretinoin, or isotretinoin alone. At 2 years, the event-free survival rate, the primary endpoint, was 66% with the dinutuximab regimen and 46% with isotretinoin alone (p<0.01). The overall survival rate was 86% with the dinutuximab regimen compared to 75% with isotretinoin alone (p<0.02).2 The recommended dose of dinutuximab is 17.5 mg/m² daily infused IV over 10-20 hours for 4 consecutive days for up to 5 cycles. Dinutuximab can cause life-threatening infusion reactions, severe pain requiring treatment with IV opioids, peripheral neuropathy, capillary leak syndrome, visual disturbances, hemolytic-uremic syndrome, and other serious adverse effects. The cost for one 17.5 mg single-use vial is $7,500.3

1. S Dhillon. Dinutuximab: first global approach. Drugs 2015; 75: 923.
2. AL Yu et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med 2010; 363:1324.
3. Approximate WAC. WAC = wholesaler acquisition cost, or manufacturer's published price to wholesalers; WAC represents published catalogue or list prices and may not represent an actual transactional price. Source: AnalySource® Monthly. March 5, 2016. Reprinted with permission by First Databank, Inc. All rights reserved. ©2016. www.fdbhealth.com/policies/drug-pricing-policy.

Download complete U.S. English article

The FDA has approved nivolumab (Opdivo – BMS), an IV programmed death receptor-1 (PD-1) blocking antibody, for treatment of unresectable or metastatic melanoma that has progressed following treatment with ipilimumab (and a BRAF inhibitor in patients who are BRAF V600 mutation positive) and for treatment of metastatic squamous non-small cell lung cancer (NSCLC) that has progressed on or after platinum-based chemotherapy. It is the second PD-1 inhibitor to be marketed in the US after pembrolizumab (Keytruda), and the first to be approved for treatment of NSCLC.

The FDA has approved pembrolizumab (Keytruda – Merck), a human programmed death receptor-1 (PD-1) blocking antibody, for treatment of unresectable or metastatic melanoma that has progressed following treatment with ipilimumab (Yervoy) and, if the patient is BRAF V600 mutation positive, a BRAF inhibitor. It is the fi rst PD-1 inhibitor to be marketed in the US. Nivolumab, another PD-1 inhibitor, is available in Japan. Pembrolizumab was previously known as lambrolizumab.

The FDA has approved vemurafenib (Zelboraf – Genentech), a kinase inhibitor, for treatment of unresectable or metastatic melanoma with the BRAF V600E mutation, which is found in 30-60% of melanomas. An FDA-approved test can detect the mutation in DNA from melanoma tissue.

The FDA has approved ipilimumab (Yervoy – Bristol-Myers Squibb), a recombinant human monoclonal antibody, for treatment of unresectable or metastatic melanoma.

Anakinra (Kineret - Amgen), an interleuken-1 (IL-1) receptor antagonist, has been approved by the FDA for treatment of moderately to severly active rheumatoid arthritis in adults who have failed at least one disease-modifying anti-rheumatic drug(DMARD) such as methotrexate (Medical Letter 2000; 24:57).