FDA Approves Enzalutamide for Nonmetastatic Castration-Sensitive Prostate Cancer


The approval makes enzalutamide the first and only androgen receptor-signaling inhibitor approved for the treatment of nonmetastatic castration-sensitive prostate cancer with biochemical recurrence at high risk for metastatic.

The FDA has approved a supplemental new drug (sNDA) application for enzalutamide (Xtandi; Astellas Pharma Inc, Pfizer Inc), making the drug the first and only androgen receptor signaling inhibitor approved for the treatment of individuals with nonmetastatic castration-sensitive prostate cancer (nmCSPC) with biochemical recurrence at high risk for metastatic (high-risk BCR), according to a press release.1

3d rendered illustration of the anatomy of a cancer cell | Image Credit: SciePro - stock.adobe.com

SciePro - stock.adobe.com

The sNDA also received priority review designation, fast track designation, and real-time oncology review, based on results from the phase 3 EMBARK trial (NCT02319837). According to the press release, those with nmCSPC with high-risk BCR could be treated with enzalutamide with or without a gonadotropin-releasing hormone analog therapy.1

“Having had the privilege of taking care of patients with prostate cancer for nearly 40 years, I have been fortunate to have participated in many of the prostate cancer landscape changing trials; notably, we have not progressed our evidenced-based care for patients with BCR, also known as nmCSPC, until the completion of the EMBARK trial,” Neal Shore, MD, FACS, chief medical officer of Strategic Innovation and Pharmacy at GenesisCare USA, said in a statement. “Previously, treatment options for these BCR patients, especially those who have a high likelihood of developing metastases were limited. The FDA approval of [enzalutamide] for patients with nmCSPC with BCR at high risk of metastasis represents an important advancement whereby an androgen deprivation signaling inhibitor, enzalutamide, has achieved standard of care discussion for patient-physician decision-making.”1

The EMBARK trial compared enzalutamide plus leuprolide, the placebo plus leuprolide, and enzalutamide as a single agent for individuals with nonmetastatic hormone-sensitive prostate cancer (nmHSPC) or nmCSPC with high-risk BCR. There were 1068 individuals enrolled at sites in the United States, Canada, Europe, South America, and the Asia-Pacific region, according to the press release.1

About The EMBARK Trial

Trial Name: Safety and Efficacy Study of Enzalutamide Plus Leuprolide in Patients With Nonmetastatic Prostate Cancer

Clinical Trails.gov ID: NCT02319837

Sponsor: Pfizer

Completion Date: January 2023

The trial met its primary endpoints of metastasis-free survival, defined as the time between randomization and the earliest objective evidence of radiographic progression, confirmed with central imaging, or death by any cause, in the enzalutamide plus leuprolide arm. The results showed that there was a statically significant reduction in risk of metastasis and death compared to the placebo and leuprolide, according to the press release.1

Furthermore, the study also met its key secondary endpoints, which included a statistically significant reduction in metastatic or death for enzalutamide as a single agent compared to the placebo plus leuprolide.1

Grade 3 or higher adverse events (AEs) were reported in approximately 46% of those treated with enzalutamide and leuprolide, 50% of those treated with enzalutamide as a single agent, and 43% treated with the placebo and leuprolide. Discontinuation due to AEs was reported in 21%, 18%, and 10%, respectively, according to the press release.1

Pharmacy Times Oncology Edition Call For Papers

Pharmacy Times Oncology Edition Call For Papers

“Today’s FDA approval is the culmination of over a decade of research and development as we’ve worked to bring [enzalutamide] forward for as many patients with prostate cancer as possible who may benefit,” Ahsan Arozullah, MD, MPH, senior vice president and head of Oncology Development at Astellas, said in the press release. “We are proud that [enzalutamide] can now be offered to a subset of men with [nmCSPC] with [BRC] and at high risk for metastases.”1

Enzalutamide is currently being evaluated by other regulatory agencies, including the European Medicines Agency, for an expanded indication in nmHSPC or nmCSPC with high-risk BCR due to the results of the study.1 The full results were published in The New England Journal of Medicine.1,2


  1. Pfizer and Astellas’ Xtandi approved by US FDA in earlier prostate cancer treatment setting. News release. Astellas Pharma. November 16, 2023. Accessed November 17, 2023. https://www.astellas.com/en/news/28626
  2. Freedland SJ, de Almeida Luz M, De Giorgi U, Gleave M, et al. Improved Outcomes with Enzalutamide in Biochemically Recurrent Prostate Cancer. N Engl J Med. 2023;389(16):1453-1465. doi:10.1056/NEJMoa2303974
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