PARIS– Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) have recommended approval of Cabometyx as a monotherapy for the treatment of adult patients with locally advanced or metastatic differentiated thyroid carcinoma (DTC), refractory or not eligible to radioactive iodine who have progressed during or after prior systemic therapy.
Jaume Capdevila, M.D. PhD Medical Oncologist at the Vall d´Hebron University Hospital and Vall d’Hebron Institute of Oncology (VHIO), Barcelona, and a trial investigator, said “Currently, for people living with radioactive iodine-refractory differentiated thyroid cancer, there are no standard-of-care treatment options should the cancer progress after first-line therapy. As a practicing physician regularly seeing people living with this uncommon form of cancer, I am encouraged to see the potential Cabometyx may bring for these patients with so few options.”
The CHMP positive opinion was based on results from the pivotal COSMIC-311 Phase III trial in which, at a planned interim analysis with a median follow-up of 6.2 months, Cabometyx demonstrated a significant reduction in the risk of disease progression or death by 78% versus placebo (hazard ratio [HR]: 0.22; 96% confidence interval [CI]: 0.13-0.36; p<0.0001).1 Another primary endpoint, the objective response rate (ORR), also favoured Cabometyx with 15% vs. 0% for placebo (p=0.028) at a median follow-up of 8.9 months, but did not meet the criteria for statistical significance. A further analysis, with a median follow-up of 10.1 months, was presented at the European Society for Medical Oncology (ESMO) Virtual Congress 2021, whereby Cabometyx continued to demonstrate superior median progression-free survival of 11.0 versus 1.9 months and a maintained reduction in the risk of disease progression or death of 78% versus placebo (HR: 0.22, 96% confidence interval [CI]: 0.15-0.32; p<0.0001).2 The safety profile identified in the COSMIC-311 trial across the two analyses was consistent with that previously observed for Cabometyx, and adverse events were managed with dose modifications.1,2
Steven Hildemann, M.D. PhD, Executive Vice President, Chief Medical Officer, Head of Global Medical Affairs and Global Patient Safety at Ipsen, said “With the promising interim results from the COSMIC-311 trial further reinforced by the maintained significant progression-free survival benefit demonstrated in the final analysis, we are pleased that the CHMP has concluded that Cabometyx may offer an important treatment option for people affected by this uncommon cancer. Following this positive opinion, we look forward to receiving the final decision from the European Commission, potentially bringing Cabometyx one step closer to reaching a patient population in critical need of new treatment options.”
This positive CHMP opinion follows the U.S. Food and Drug Administration’s approval in September 2021 of Cabometyx for the treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic DTC that has progressed following prior vascular endothelial growth factor receptor targeted therapy and who are radioactive iodine-refractory or ineligible.
