Cancer Treatment Today

On May 17, 2024 – Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; announced that
it has received supplemental approvals for BRAFTOVI® (generic name: encorafenib) Capsule (“BRAFTOVI”), a BRAF inhibitor, and MEKTOVI® (generic name: binimetinib) Tablet (“MEKTOVI”), a MEK inhibitor, when used in combination, in
Japan for the two new indications of “unresectable thyroid cancer with a BRAF mutation that has
progressed following chemotherapy”, and “unresectable anaplastic thyroid cancer with a BRAF
mutation”.
These approvals are based on the results of a Phase 2 study (ONO-7702/7703-03), conducted in
Japan in 22 patients with unresectable BRAFV600-mutant thyroid cancer, including 5 patients with
anaplastic thyroid cancer. The study met its primary endpoint of objective response rate (ORR) as
assessed by the Independent Central Review in the overall patient population, which was 54.5%
(12/22 cases, 95% confidence interval: 32.2 – 75.6%) in the combination therapy of BRAFTOVI and
MEKTOVI. The safety profile of the combination therapy of BRAFTOVI and MEKTOVI in the study
was consistent with those previously reported in the clinical trials of BRAFTOVI and MEKTOVI.
About Phase 2 study (ONO-7702/7703-03)
The study is a multi-centre, open-label, uncontrolled Phase 2 study (ONO-7702/7703-03),
conducted in Japan, evaluating the efficacy and safety of the combination therapy of BRAFTOVI and
MEKTOVI in patients with unresectable BRAF V600
-mutant thyroid cancer. Patients received the
combination therapy with BRAFTOVI 450 mg once daily, and MEKTOVI 45 mg twice daily, until it
was determined that treatment could not be given due to disease progression or safety reasons. The primary endpoint of the study was objective response rate (ORR) as assessed by the Independent Central Review. Secondary endpoints include ORR as assessed by physician of each medical institutes, disease control rate (DCR), overall survival (OS) and progression-free survival (PFS). In the USA this would be off-label.