Eli Lilly Advances Ovarian Cancer Drug With FDA Breakthrough

Global drugmaker Eli Lilly and Company has secured FDA Breakthrough Therapy designation for sofetabart mipitecan, offering new momentum for patients battling platinum-resistant ovarian cancer, one of the hardest-to-treat gynecologic cancers today.

The decision applies to adults with platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer who were previously treated with bevacizumab and mirvetuximab soravtansine.

The experimental therapy is a next-generation folate receptor alpha (FRα) antibody-drug conjugate designed to deliver an exatecan payload directly to tumor cells. The FDA Breakthrough Therapy designation is meant to speed development of treatments that show early signs of meaningful benefit for serious diseases with few options.

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“Platinum-resistant ovarian cancer remains one of the most challenging settings in gynecologic oncology, with limited treatment options and poor outcomes for patients,” said Bhavana Pothuri, professor at NYU Grossman School of Medicine and director of the Clinical Trials Office at Perlmutter Cancer Center.

Jacob Van Naarden, executive vice president and president of Lilly Oncology, said, “We are pleased the FDA has granted Breakthrough Therapy designation for sofetabart mipitecan…we've initiated our Phase 3 FRAmework-01 trial with the goal of bringing a potential therapeutic option to patients with advanced ovarian cancer, across all levels of folate receptor expression.”

The designation is backed by Phase 1a/b data shared at ASCO 2025 and updated at ESMO 2025. Results showed tumor responses across all FRα levels, including patients who progressed after mirvetuximab. The drug also showed a favorable safety profile, with low rates of lung toxicity, nerve damage, hair loss, and no major eye-related issues.

Lilly has now moved the drug into the global Phase 3 FRAmework-01 trial, testing it alone in platinum-resistant disease and in combination with bevacizumab for platinum-sensitive patients, in collaboration with ENGOT, the GOG Foundation, and APGOT.