Exelixis (EXEL) completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Cabometyx® (cabozantinib) tablets for previously treated patients with advanced hepatocellular carcinoma (HCC).
The sNDA submission is based on results from the CELESTIAL randomized pivotal phase 3 trial of Cabometyx in patients with advanced HCC who received prior sorafenib.
Some History: On October 16, 2017, Exelixis announced that the independent data monitoring committee for the study recommended that the CELESTIAL trial be stopped for efficacy following review at the second planned interim analysis, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with previously treated advanced HCC (pre-specified critical p-value ≤ 0.021).
The FDA, had granted orphan drug designation to cabozantinib for the treatment of advanced HCC in March 2017.
Incidence: Liver cancer is the second-leading cause of cancer death worldwide, accounting for more than 700,000 deaths and nearly 800,000 new cases each year.1 In the U.S., the incidence of liver cancer has more than tripled since 1980.2 Hepatocellular carcinoma (HCC) is the most common form of liver cancer, making up about three-fourths of the estimated nearly 42,000 new cases in the U.S. in 2018.2 HCC is the fastest-rising cause of cancer-related death in the U.S.
Without treatment, patients with advanced HCC usually survive less than 6 months.
Cabometyx tablets are approved in the United States for advanced renal cell carcinoma (RCC) and in the European Union, Norway, Iceland, Australia, Switzerland and South Korea for advanced RCC in adults who have received prior vascular endothelial growth factor (VEGF)-targeted therapy.
In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. Accordingly, Ipsen submitted to European Medicines Agency (EMA) the regulatory dossier for cabozantinib as a treatment for first-line advanced RCC in the European Union on August 28, 2017; on September 8, 2017, Ipsen announced that the EMA validated the application.
In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan, including RCC.
Cabometyx is not indicated for previously treated advanced HCC.
For Important Safety Information and full U.S. prescribing information please go to the firm’s press release and at https://cabometyx.com/downloads/CABOMETYXUSPI.pdf.
The submission of the supplemental New Drug Application (sNDA) to the FDA for Cabometyx® tablets to treat previously treated patients with advanced hepatocellular carcinoma (HCC) opens the door to another important large market for Cabometyx. The good news is that the independent committee decided to halt the trials after the product demonstrated statistically significant and clinically meaningful improvement in overall survival (OS) compared with placebo.
This submission is not the last regarding the number of cancers that Cabometyx is expected to treat.
This is good news.
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