Health Canada approves Ipsen’s CABOMETYX® (cabozantinib) for Patients with Previously Treated Radioactive Iodine-Refractory or Ineligible Differentiated Thyroid Cancer

Pivotal phase III trial data showed CABOMETYX^® demonstrated a significant improvement in progression-free survival.¹

Mississauga, ON – May 4, 2022 – Ipsen Biopharmaceuticals Canada Inc. today announced the Health Canada approval of CABOMETYX^® (cabozantinib) for the treatment of adults with locally advanced or metastatic differentiated thyroid carcinoma (DTC) that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory (RAI-R) or ineligible.¹ CABOMETYX^® was granted priority review for DTC by Health Canada because of the significant unmet need it fills for this patient population.

“Patients living with RAI-R DTC have limited treatment options, and often go without second line treatment even though they are well enough to receive one,” said Dr. Sebastien Hotte, medical oncologist, the Juravinski Cancer Centre, Associate Professor, McMaster University. “The progression-free survival benefit demonstrated in the phase III COSMIC-311 trial means CABOMETYX^® is of significant importance for patients I see every day. Thanks to this approval, patients who thought they had run out of options may have a chance for more time without disease progression.”

It is estimated that 6,700 Canadians were diagnosed with thyroid cancer in 2021, with DTC accounting for up to 95% of cases.^2,3 The incidence of thyroid cancer is three times higher in women than men and is the ninth most common cancer in the world.⁴ Most commonly treated with surgery, followed by ablation of the remaining thyroid tissue with RAI, approximately 5 to 15% of DTC cases become refractory to RAI treatment.⁵ Patients who develop RAI-refractory DTC have a poor prognosis with an average estimated survival of three to five years.⁶

This approval is based on the Phase III COSMIC-311 trial, which demonstrated that CABOMETYX^® showed statistically significant improvement in progression-free survival for patients vs placebo. In the primary analysis conducted after median follow-up of 6.2 months, CABOMETYX^® was associated with a 78% reduction in risk of disease progression or death versus placebo (hazard ratio [HR]: 0.22; 96% confidence interval [CI]: 0.13-0.36; p>0.0001).¹

“We’re proud to make new treatment options like CABOMETYX^® available for patients suffering from DTC who previously had no other options after progression on a VEGF therapy,” said Ed Dybka, General Manager, Ipsen Biopharmaceuticals Canada Inc. “With this approval, CABOMETYX^® now has five indications, underscoring Ipsen’s commitment to addressing unmet patient needs and the potential for CABOMETYX^® to improve the lives of patients and their families.”

About the COSMIC-311 trial

COSMIC-311 is a randomized, double-blind, placebo-controlled Phase III trial that enrolled 258 patients with locally advanced or metastatic differentiated thyroid cancer (papillary thyroid carcinoma or follicular thyroid carcinoma) that had progressed following prior VEGFR-targeted therapy and were radioactive iodine (RAI)-refractory or ineligible.¹ Patients were randomized into a 2:1 ratio to receive either CABOMETYX^® 60 mg or placebo once-daily.¹ The primary endpoints were progression-free survival (PFS) in the intention-to-treat population as well as objective response rate (ORR) in the first 100 randomly assigned patients (objective response rate intention-to-treat [OITT] population), both evaluated by a blinded independent radiology committee. Additional endpoints include safety, overall survival and quality of life.⁷ Exelixis is the sponsor of COSMIC-311, and Ipsen is co-funding the trial. More information about this trial is available at ClinicalTrials.gov.

About CABOMETYX^® (cabozantinib)

CABOMETYX^® is the first multi-targeted tyrosine kinase inhibitor (TKI) to target VEGFR, MET  and AXL pathways that are involved in tumour progression. CABOMETYX^® is a multi-kinase inhibitor. It works by blocking the action of proteins called receptor tyrosine kinases (RTKs). RTKs are involved in cell growth and the development of new blood vessels. These proteins can be present in high amounts in cancer cells. By blocking their action, CABOMETYX^® can slow down how fast the tumour grows, help to block the blood supply that the cancer needs and may increase the length of time before the cancer gets worse.¹

CABOMETYX^® has three indications for the treatment of patients with advanced renal cell carcinoma (RCC) and one indication for the treatment of patients with advanced hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.

About Ipsen in North America

Ipsen is a global biopharmaceutical company focused on innovation and specialty care. At Ipsen, we focus our resources, investments and energy on discovering, developing and commercializing medicines in three key therapeutic areas – Oncology, Rare Disease and Neuroscience. Our North American operations are located in Cambridge, Massachusetts, one of the company’s three global hubs. Based in the heart of Kendall Square, our fully integrated biopharmaceutical business includes Commercial, Research & Development, Manufacturing, and Business Development. Combined with our Canadian headquarters in Mississauga, Ontario, and other locations, we employ approximately 600 people in North America. For more information please visit www.ipsen.com/canadaEN.

For further information:

1. CABOMETYX^® Product Monograph. Ipsen Biopharmaceuticals Canada Inc. 27 April 2022
2. Canadian Cancer Society. Thyroid Cancer Statistics. https://cancer.ca/en/cancer-information/cancer-types/thyroid/statistics#:~:text=Incidence%20and%20mortality&text=6%2C700%20Canadians%20will%20be%20diagnosed,130%20will%20die%20from%20it. Last accessed April 26, 2022.
3. Cancer.Net. ASCO. Thyroid Cancer: Introduction. https://www.cancer.net/cancer-types/thyroid-cancer/introduction. Last accessed April 26, 2022.
4. Sung. H et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries: CA: A Cancer Journal for Clinicians. Doi: 10.3322/caac.21660.
5. Worden F. Treatment strategies for radioactive iodine-refractory differentiated thyroid cancer. Ther Adv Med Oncol. 6:267–279. doi: 10.1177/1758834014548188.
6. Fugazzola L. et al. 2019 European Thyroid Association Guidelines for the Treatment and Follow-Up of Advanced Radioiodine-Refractory Thyroid Cancer. Eur Thyroid J. 2019;8:227–245. doi: 10.1159/000502229.
7. Brose et al., Cabozantinib for radioiodine-refractory differentiated thyroid cancer (COSMIC-311): a randomized, double-blind, placebo-controlled, phase 3 trial. Lancet Oncology. 2021; 22:8.