- Exelixis is eligible to receive a milestone payment of $40 million for the approval of the second-line treatment of HCC
- In May of 2018, the FDA accepted for filiing an sNDA for Cabometyx for previously treated advanced HCC
- A Prescription Drug User Fee Act (PDUFA) action date of January 14 was assigned
Exelixis, Inc. (NASDAQ:EXEL) today announced that its partner Ipsen received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), for Cabometyx (cabozantinib) tablets as a monotherapy for the treatment of hepatocellular carcinoma (HCC) in adults who have been previously treated with sorafenib. The positive CHMP opinion will now be reviewed by the European Commission, which has the authority to approve medicines for the European Union.
“This positive CHMP opinion represents significant progress for patients in Europe with this aggressive form of liver cancer who progress on prior systemic therapy, a large underserved patient population that currently only has one approved second-line treatment option,” said Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis. “We are excited about the potential therapeutic benefits Cabometyx may offer the liver cancer community and look forward to the European Commission’s decision.”
Under the terms of the Collaboration Agreement with Ipsen, Exelixis is eligible to receive a milestone payment of $40 million for the approval of the second-line treatment of HCC. This milestone would be paid by Ipsen within 70 days of the approval decision by the European Commission.
Cabometyx is currently approved in the European Union for the treatment of advanced renal cell carcinoma (RCC) in adults who have received prior VEGF-targeted therapy and for previously untreated intermediate- or poor-risk advanced RCC. The CHMP recommendation to expand the indication is based on results from the CELESTIAL trial of Cabometyx in patients with advanced HCC who received prior sorafenib. In this phase 3 pivotal trial, Cabometyx demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo.
On May 29, 2018, Exelixis announced that the U.S. Food and Drug Administration (FDA) accepted for filing the supplemental New Drug Application (sNDA) for Cabometyx for previously treated advanced HCC and assigned a Prescription Drug User Fee Act (PDUFA) action date of January 14, 2019. An sNDA is an application to the FDA that, if approved, will allow a drug sponsor to make changes to a previously approved product label, including modifications to the indication.
Liver cancer is the second-leading cause of cancer death worldwide, accounting for more than 700,000 deaths and 800,000 new cases each year.2 In the U.S., the incidence of liver cancer has more than tripled since 1980.