Type of information: Refusal of a Market Autorisation in the EU
Product name: Cabometyx®
Therapeutic area: Cancer - Oncology
- tyrosine kinase inhibitor. Cabozantinib is a kinase inhibitor that blocks abnormal kinase proteins involved in the development and growth of medullary cancer cells. Cabozantinib inhibits the activity of tyrosine kinases including MET, VEGFRs and AXL. These receptor tyrosine kinases are involved in both normal cellular function and in pathologic processes such as oncogenesis, metastasis, tumor angiogenesis, and maintenance of the tumor microenvironment. In April 2015, cabozantinib received Fast Track designation by the FDA for the potential treatment of advanced renal cell carcinoma patients who have received one prior therapy. Exelixis and Ipsen jointly announced an exclusive licensing agreement for the commercialization and further development of cabozantinib.
- On April 25, 2016 Cabometyx™ was approved by the FDA for the treatment of patients with advanced renal cell carcinoma who have received prior anti-angiogenic therapy.
Company: Exelixis (USA - CA) Ipsen (France)
Disease: previously untreated advanced renal cell carcinoma
- • On March 22, 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive
opinion recommending a change to the terms of the marketing authorisation for Cabometyx®. The marketing authorisation holder for this medicinal product is Ipsen Pharma. The CHMP adopted an extension to the existing indication as follows:
- “Cabometyx® is indicated for the treatment of advanced renal cell carcinoma :
- • in treatment-naïve adults with intermediate or poor risk
- • in adults following prior vascular endothelial growth factor (VEGF)-targeted therapy.”
- • On September 8, 2017, Ipsen announced that
the European Medicines Agency (EMA) has validated the
application for variation to the Cabometyx® (cabozantinib) marketing authorization for the addition of a new indication in first-line treatment of advanced renal cell carcinoma.
The filing is based on the results of CABOSUN, a phase II trial demonstrating that Cabometyx® prolongs progression free survival in treatment-naive patients with intermediate- or poor-risk advanced renal cell carcinoma compared to sunitinib, the standard of care for more than 10 years.
- • On August 16, 2017, Exelixis announced it has completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Cabometyx® (cabozantinib) tablets as a treatment for patients with previously untreated advanced renal cell carcinoma. The sNDA submission is based on results from the CABOSUN randomized phase 2 trial of Cabometyx® in patients with previously untreated advanced renal cell carcinom with intermediate- or poor-risk disease per the International Metastatic Renal Cell Carcinoma Database Consortium (IMDC).
Submission of marketing authorization application USA : 2017-08-16
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
Favourable opinion UE: 2018-03-22
Favourable opinion USA:
Orphan status USA:
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: