On April 13, 2021, the U.S. Food and Drug Administration (FDA) granted accelerated approval to sacituzumab govitecan (Trodelvy®) for patients with locally advanced or metastatic urothelial cancer  who previously received a platinum-containing chemotherapy and either a programmed death receptor-1 (PD-1) or a programmed death-ligand 1 (PD-L1) inhibitor.

FDA Grants Accelerated Approval to Sacituzumab Govitecan for Advanced Urothelial Cancer

Efficacy and safety were evaluated in a single-arm, multicenter trial (TROPHY IMMU-132-06; NCT03547973) of 112 patients with locally advanced or metastatic urothelial cancer who received prior treatment with a platinum-containing chemotherapy and either a PD-1 or PD-L1 inhibitor. Patients received sacituzumab govitecan 10 mg/kg via IV infusion on days 1 and 8 of a 21-day treatment cycle.

The main efficacy endpoints were objective response rate (ORR) and duration of response (DOR), evaluated by independent review using response evaluation criteria in solid tumors 1.1. Confirmed ORR was 27.7% (95% CI = 19.6, 36.9) with 5.4% complete responses and 22.3% partial responses. Median DOR was 7.2 months (n = 31; 95% CI = 4.7, 8.6; range = 1.4+, 13.7).

The most common adverse reactions (> 25%) were neutropenia, nausea, diarrhea, fatigue, alopecia, anemia, vomiting, constipation, decreased appetite, rash, and abdominal pain.

The recommended sacituzumab govitecan dose is 10 mg/kg once weekly on days 1 and 8 of 21-day treatment cycles until patients experience disease progression or unacceptable toxicity.

View full prescribing information for sacituzumab govitecan.

FDA approved the indication under accelerated approval based on tumor response rate and DOR. Continued approval for the indication is contingent on verification and description of clinical benefit in a confirmatory trial.

The review used the Real Time Oncology Review pilot program, which streamlined data submission prior to the filing of the entire clinical application, and Assessment Aid, a voluntary submission from the applicant to facilitate FDA’s assessment. FDA approved the application six weeks ahead of its goal date.

The application was granted priority review and fast track designation. 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 they suspect are associated with the use of any medicine or 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