In fiscal year 2011, FDA approved 7 “new molecular entities” (NMEs) in oncology.
5. Adcetris (brentuximab vedotin) to treat two types of lymphoma.
Importance: Adcetris is the first new FDA approved treatment for Hodgkin’s lymphoma (HL) since 1977, and the first specifically indicated to treat a rare lymphoma known as systemic anaplastic large cell lymphoma (ALCL). These cancers are orphan diseases. The National Cancer Institute estimates that 8,830 new cases of HL will be diagnosed in the
in 2011 and about 1,300 people will die from the disease. Systemic ALCL is a rare malignant tumor (non-Hodgkin’s lymphoma) that may appear in several parts of the body including the lymph nodes, skin, bones, soft tissue, lungs or liver. United States
Actions to speed drug testing and review: Adcetris was given a Fast Track designation for its potential to address an unmet medical need in patients with Hodgkin’s lymphoma. The effectiveness of Adcetris in patients with HL was evaluated in a single clinical trial involving 102 patients. In the single-arm trial, patients were only treated with Adcetris. FDA did not require a concurrent control group or second trial to replicate the results. The study’s primary endpoint was objective response rate—the percentage of patients who experienced complete or partial cancer shrinkage or disappearance after treatment—rather than improved survival. Seventy-three percent of patients achieved either a complete or partial response to the treatment. On average, these patients responded to the therapy for 6.7 months. The effectiveness of Adcetris in patients with systemic ALCL was evaluated in a single clinical trial in 58 patients. In the single-arm trial, patients were only treated with Adcetris. FDA did not require a concurrent control group or second trial to replicate the results. Similar to the HL trial, the trial’s primary endpoint was objective response rate. Of the patients receiving Adcetris for ALCL, 86 percent experienced either a complete or partial response and responded on average for 12.6 months. Adcetris was reviewed under the FDA’s priority review program, which provides for an expedited six-month review of drugs, and was approved under FDA’s accelerated approval program, which allowed approval of the drug based on surrogate endpoints (tumor shrinkage) rather than a demonstration of improved survival. The data on safety and effectiveness were limited because of the small number of patients tested and the lack of a control group, but FDA granted accelerated approval with a commitment from the sponsor to provide additional data after approval.
Safety issues: FDA concluded that the benefits of Adcetris outweighed the risk of reported side effects, including a decrease in infection fighting white blood cells (neutropenia), nerve damage (peripheral sensory neuropathy), fatigue, nausea, anemia, upper respiratory infection, diarrhea, fever, cough, vomiting, and low blood platelet levels (thrombocytopenia). Pregnant women should be aware that Adcetris might cause harm to their unborn baby.
Source: FDA - FY 2011 Innovative Drug Approvals