On December 18, 2020, the U.S. Food and Drug Administration (FDA) approved the first oral gonadotropin-releasing hormone receptor antagonist, relugolix (Orgovyx™), for adult patients with advanced prostate cancer. 

Efficacy was evaluated in a randomized, open-label trial (HERO; NCT03085095) that required at least one year of androgen deprivation therapy with either prostate cancer recurrence following radiation or surgery or newly diagnosed castration-sensitive advanced prostate cancer. Patients (N = 934) were randomized two to one to receive an oral loading dose of 360 mg of relugolix on the first day followed by daily oral doses of 120 mg or subcutaneous injection of 22.5 mg of leuprolide acetate every three months for 48 weeks. 

The main efficacy outcome measure was medical castration rate defined as achieving and maintaining serum testosterone suppression to castrate levels (< 50 ng/dL) by day 29 through 48 weeks of treatment. The medical castration rate was 96.7% (95% CI = 94.9%, 97.9%) in the relugolix arm. 

The most common adverse reactions (≥ 10%) in patients receiving relugolix were hot flush, musculoskeletal pain, fatigue, diarrhea, and constipation. The most common laboratory abnormalities (≥ 15%) were increased glucose, triglycerides, alanine aminotransferase, and aspartate aminotransferase. Decreased hemoglobin was also observed. 

The recommended relugolix dose is a loading dose of 360 mg on the first day followed by a daily dose of 120 mg at approximately the same time. 

View full prescribing information for relugolix

The review used Assessment Aid, a voluntary submission from the applicant to facilitate FDA’s assessment. 

FDA granted the application priority review. A description of FDA expedited programs is in the Guidance for Industry—Expedited Programs for Serious Conditions—Drugs and Biologics.   

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.         

For assistance with single-patient oncology investigational new drug applications, contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.