Cabozantinib Receives U.S. FDA Approval as a First-Line Treatment in Combination with Nivolumab for Advanced Renal Cell Carcinoma

– FDA approval based on CheckMate -9ER trial, in which the combination of CABOMETYX and OPDIVO significantly improved overall survival while doubling progression-free survival and objective response rate versus sunitinib as a first-line treatment for patients with advanced RCC –

– Exelixis prepared to fully support expanded indication immediately –

– Application approved prior to Prescription Drug User Fee Act action date of February 20, 2021, and reviewed under the Real-Time Oncology Review pilot program –


San Francisco, CA (UroToday.com) -- Exelixis, Inc. announced that the U.S. Food and Drug Administration (FDA) approved CABOMETYX® (cabozantinib) for patients with advanced renal cell carcinoma (RCC) as a first-line treatment in combination with OPDIVO® (nivolumab). RCC is the most common form of kidney cancer, which is among the 10 most frequently diagnosed cancers in the U.S. annually.1


“This combination of cabozantinib and nivolumab significantly improved key efficacy measures compared to sunitinib – progression-free survival, overall survival, and objective response rate – while showing a low rate of treatment discontinuations due to side effects. The therapeutic benefit demonstrated in CheckMate -9ER and quality of life measures explored emphasize the role of this combination for patients with advanced kidney cancer,” said Dr. Toni Choueiri, Director of the Lank Center for Genitourinary Oncology at Dana-Farber Cancer Institute and the Jerome and Nancy Kohlberg Professor of Medicine at Harvard Medical School. “With this important FDA approval, the combination is poised to become a standard in newly diagnosed metastatic kidney cancer.”
The approval is based on results from CheckMate -9ER, a phase 3 pivotal trial evaluating the combination of CABOMETYX and OPDIVO compared with sunitinib in previously untreated advanced or metastatic RCC. These results were presented during the European Society of Medical Oncology Virtual Congress 2020 in September. The FDA reviewed the application for CABOMETYX and OPDIVO under the Real-Time Oncology Review (RTOR) pilot program and Fast Track designation. The RTOR pilot program, which allows an applicant to pre-submit components of the application to allow the FDA to review clinical trial data before the complete filing is submitted, aims to explore a more efficient review process to ensure safe and effective treatments are available to patients sooner.

“As the only combination treatment regimen to double median progression-free survival and objective response rate compared with sunitinib while also significantly improving overall survival, we are excited that CABOMETYX in combination with OPDIVO is now available for the first-line treatment of patients with advanced kidney cancer,” said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. “This approval is a meaningful milestone for this patient community and speaks to the broad potential of CABOMETYX as we continue to generate important clinical trial results supporting its use in combination with immune checkpoint inhibitors to benefit patients with other difficult-to-treat cancers. We would like to thank the clinical trial participants, the physicians and their staff who participated in the CheckMate -9ER trial and to acknowledge the team at the FDA for their collaboration during the review of our application.”

In CheckMate -9ER, the combination regimen significantly improved overall survival (OS) compared with sunitinib (HR= 0.60, 98.89% CI 0.40-0.89; p=0.001). Median OS has not yet been reached in either treatment arm. Median progression-free survival (PFS) was doubled at 16.6 months for CABOMETYX in combination with OPDIVO compared with 8.3 months for sunitinib (HR 0.51, 95% CI 0.41-0.64; p<0.0001). Objective response rate (ORR) was also doubled: 56% with CABOMETYX in combination with OPDIVO and 27% with sunitinib (p<0.0001). Consistent results for PFS were observed across subgroups of International Metastatic RCC Database Consortium risk status and PD-L1 tumor expression with CABOMETYX in combination with OPDIVO.

CABOMETYX in combination with OPDIVO was generally well tolerated and reflected the known safety profiles of the tyrosine kinase inhibitor and immunotherapy components in previously untreated advanced RCC. The most common adverse reactions reported in at least 20% of patients treated with CABOMETYX in combination with OPDIVO were diarrhea, fatigue, hepatotoxicity, palmar-plantar erythrodysesthesia, stomatitis, rash, hypertension, hypothyroidism, musculoskeletal pain, decreased appetite, nausea, dysgeusia, abdominal pain, cough, and upper respiratory tract infection. The discontinuation rate due to all causality adverse events in the CABOMETYX in combination with OPDIVO arm was 20% for either CABOMETYX or OPDIVO (8% for CABOMETYX only, 7% for OPDIVO only and 6% for both CABOMETYX and OPDIVO due to the same adverse event at the same time).

“While significant progress has been made in the treatment landscape for advanced kidney cancer over the last several years, patients still need more therapeutic options to treat this disease as we search for a possible cure,” said Bryan Lewis, President, and Co-founder of KidneyCAN. “As patients are living longer with advanced kidney cancer, focusing on the safety and effectiveness of new treatments has become even more important. The findings for the combination of CABOMETYX and OPDIVO in the CheckMate -9ER trial make the FDA approval of this combination a notable development for the patient community.”

Exelixis’ partner Ipsen Pharma SAS (Ipsen), which has exclusive rights to commercialize and develop CABOMETYX outside of the U.S. and Japan, and Bristol-Myers Squibb Company (BMS) each submitted type II variation applications for CABOMETYX in combination with OPDIVO to the European Medicines Agency (EMA). On September 12, 2020, the EMA validated the type II variations, confirming the submissions are complete and beginning the EMA’s centralized review process. On October 27, 2020, Takeda Pharmaceutical Company Limited (Takeda), Exelixis’ partner responsible for the clinical development and commercialization of CABOMETYX in Japan, and Ono Pharmaceuticals Co., Ltd., BMS’ development and commercialization partner in Japan, submitted a supplemental application to the Japanese Ministry of Health, Labour and Welfare for manufacturing and marketing approval of CABOMETYX in combination with OPDIVO for the treatment of patients with unresectable, advanced or metastatic RCC.

Source: Exelixis, Inc. 2021. Exelixis Announces U.S. FDA Approval Of CABOMETYX® (Cabozantinib) In Combination With OPDIVO® (Nivolumab) As A First-Line Treatment For Patients With Advanced Renal Cell Carcinoma | Exelixis, Inc.


Related Content:

ESMO 2020: CheckMate-9ER Study of Nivolumab Combined With Cabozantinib Vs. Sunitinib in Participants With Previously Untreated mRCC - Toni Choueiri

ESMO Virtual Congress 2020: Nivolumab + Cabozantinib vs Sunitinib in First-line Treatment for Advanced Renal Cell Carcinoma: First Results From The Randomized Phase 3 CheckMate 9ER Trial