- FDA grants Breakthrough Therapy designation to sofetabart mipitecan for platinum-resistant ovarian cancer.
- Sofetabart mipitecan is a folate receptor alpha antibody-drug conjugate with proprietary linker technology.
- Phase 3 FRAmework-01 trial initiated for advanced ovarian cancer treatment.
- Initial Phase 1 results showed promising tolerability and efficacy across all folate receptor expression levels.
FDA Breakthrough Therapy Designation
The U.S. FDA has granted Breakthrough Therapy designation to sofetabart mipitecan for treating adult patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. This designation aims to expedite the development and review of drugs that show potential for substantial improvement over existing therapies.
Drug Profile
Sofetabart mipitecan is a novel folate receptor alpha (FRα) antibody-drug conjugate (ADC) that incorporates proprietary linker technology and an exatecan payload. It targets patients who have previously received bevacizumab and mirvetuximab soravtansine, if eligible.
Clinical Trials and Results
Initial Phase 1 results, presented at the 2025 ASCO Annual Meeting and updated at the 2025 ESMO Congress, demonstrated promising responses across all dose levels and FRα expression levels. The data indicated a favorable tolerability profile, with low rates of interstitial lung disease, peripheral neuropathy, and alopecia, and no significant ocular toxicity.
Phase 3 FRAmework-01 Study
Sofetabart mipitecan has advanced into the Phase 3 FRAmework-01 study, a global trial investigating its use as a monotherapy for platinum-resistant ovarian cancer and in combination with bevacizumab for platinum-sensitive ovarian cancer. This study is conducted in partnership with the European Network for Gynaecological Oncological Trial groups, the GOG Foundation, and the Asia-Pacific Gynecologic Oncology Trials Group.
