FDA Approves First Treatment for Type of Localized Prostate Cancer


The US Food and Drug Administration (FDA) has approved the first treatment for a type of localized prostate cancer, using a novel endpoint to support the approval.

Apalutamide (Erleada, Janssen Pharmaceutical Companies) is a next-generation androgen receptor inhibitor designed to treat patients with non-metastatic castration-resistant prostate cancer.

FDA approval came following data from a randomized clinical trial involving 1207 patients with such disease. Patients received either apalutamide or placebo and were also treated with hormone therapy – either gonadotropin-releasing hormone analog therapy or surgical castration. The median metastasis-free survival for patients taking apalutamide was 40.5 months compared with 16.2 months for those taking placebo. Risk of metastasis or death was also lowered by 72% for those taking apalutamide.

This FDA approval was the first to use metastasis-free survival as an endpoint. “In the trial supporting approval, apalutamide had a robust effect on this endpoint,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research (February 14, 2018). “This demonstrates the agency’s commitment to using novel endpoints to expedite important therapies to the American public.”


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Adverse events associated with apalutamide include fatigue, hypertension, rash, diarrhea, nausea, weight loss, arthralgia, falls, hot flush. Decreased appetite, fractures, and peripheral edema. Severe side effects include falls, fractures, and seizures.—Zachary Bessette