Japan’s Ministry of Health, Labour and Welfare has approved risovalisib for patients with advanced or recurrent ovarian clear cell carcinoma who carry PIK3CA mutations and whose disease has progressed after chemotherapy. The decision offers a new targeted option for a difficult-to-treat form of ovarian cancer.
Alongside the drug, the AmoyDx PIK3CA Mutation Detection Kit was also approved. This test uses real-time PCR on tumor samples to identify patients who are most likely to benefit from the treatment by detecting PIK3CA mutations.
The approval is based on results from the Phase 2 CYH33-G201 trial, which showed that risovalisib has meaningful antitumor activity with a manageable safety profile. In the study, adult women with recurrent or persistent clear cell carcinoma of the ovary, fallopian tube, or peritoneum received a daily 40 mg dose of the drug.
Patients were excluded if they had previously received similar pathway inhibitors, had active hepatitis, recent major surgery, or serious heart conditions. The trial mainly measured tumor response, along with progression-free survival, overall survival, safety, and how the drug behaves in the body.
The drug had already been granted orphan drug status in June 2025, reflecting its potential to address an unmet need in a rare cancer subtype.