Eflornithine (Iwilfin – US WorldMeds), an oral ornithine decarboxylase inhibitor, has been approved by the FDA to reduce the risk of relapse in children and with high-risk neuroblastoma who had at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy. About 40-50% of neuroblastoma cases are classified as high-risk and they account for ~15% of all pediatric cancer deaths. Eflornithine is the first drug to be approved to reduce the risk of relapse in children with high-risk neuroblastoma. Eflornithine was previously available in the US in injectable and topical formulations for other indications; both formulations were removed from the market for business reasons

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.

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The tables in this article list drugs used for treatment of cancer in the USA and Canada and their major adverse effects. The choice of drugs in Table I is based on the opinions of Medical Letter consultants. Some drugs are listed for indications for which they have not been approved by the US Food and Drug Administration. In some cases, such as elderly patients or those with many co-morbid illnesses, the regimen of choice might not be suitable. For many of the cancers listed, surgery and/or radiation therapy may be the treatment of choice or may also be part of the management. Anticancer drugs and their adverse effects are listed in Table II on page 46. A partial list of brand names appears on page 52.

The tables in this article list drugs used for treatment of cancer in the USA and Canada. The choices of drugs in Table 1 is based on the opinions of Medical Letter consultants. Some drugs are listed for indications for which they have not been approved by the US Food and Drug Administration. For many of the cancers listed, surgery and/or radiation therapy are also part of the management of the disease.