Lilly’s Sofetabart Mipitecan Wins FDA Breakthrough Tag in Tough‑to‑Treat Ovarian Cancer | iPharmaCenter
- ipharmaservices
- Jan 22
- 2 min read
Lilly has disclosed that the U.S. Food and Drug Administration has granted Breakthrough Therapy status to sofetabart mipitecan (LY4170156) for adults with platinum‑resistant epithelial ovarian, fallopian tube or primary peritoneal cancer whose disease has already been treated with bevacizumab and, where appropriate, mirvetuximab soravtansine.
The investigational therapy is an antibody-drug conjugate directed against folate receptor alpha, built with a proprietary linker and carrying an exatecan‑based cytotoxic payload.
This regulatory designation is reserved for experimental medicines targeting serious or life‑threatening conditions where early clinical findings suggest the potential for substantial improvement over currently available options on clinically meaningful outcomes. In practice, it opens the door to more intensive interaction with regulators and the possibility of an accelerated development and review pathway.
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The decision from the agency is supported by early data from an ongoing phase 1a/1b trial.
On the back of these findings, sofetabart mipitecan has moved into the pivotal FRAmework‑01 phase 3 programme (NCT07213804). This global study is evaluating the drug as a single‑agent in platinum‑resistant ovarian cancer and in combination with bevacizumab for patients with platinum‑sensitive ovarian cancer. The trial is being run in collaboration with leading academic networks including the European Network for Gynaecological Oncological Trial groups (ENGOT, with GINECO and NOGGO as lead groups), the GOG Foundation and the Asia‑Pacific Gynecologic Oncology Trials Group.
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About ovarian cancer
Ovarian cancer remains one of the most lethal malignancies affecting women in the United States, ranking among the top causes of cancer‑related death. Most patients initially benefit from platinum‑based chemotherapy, but roughly seven in ten will ultimately relapse, and the duration of response typically shortens with each subsequent line of treatment.
When the tumour returns during, or within six months of completing, a platinum‑containing regimen, the disease is categorised as platinum‑resistant, a setting where therapeutic options are limited and outcomes are poor.
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About Sofetabart mipitecan and mechanism of action
Sofetabart mipitecan consists of a humanised, Fc‑silent monoclonal antibody that selectively recognises folate receptor alpha, conjugated to the topoisomerase I inhibitor exatecan via a specially designed cleavable polysarcosine linker (PSARlink). The molecule was engineered to engage folate receptor alpha over a broad spectrum of expression levels while optimising the balance between efficacy and safety.
Folate receptor alpha itself is a cell‑surface protein encoded by FOLR1 that binds folic acid and reduced folate molecules and shuttles them into cells to support DNA synthesis, cell division and growth. It is frequently overexpressed in several solid tumours, including ovarian, certain non‑small cell lung and colorectal cancers. Sofetabart mipitecan is currently being evaluated not only in ovarian cancer but also in other folate receptor alpha‑positive solid tumours within ongoing clinical studies, including trials registered as NCT06400472 and NCT07213804.
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