Global pharma powerhouse Eli Lilly and Company has got the US FDA green light to offering a new hope for patients facing one of the most aggressive forms of ovarian cancer.

 

Yes, the pharma company has announced that the receipt of Breakthrough Therapy designation to sofetabart mipitecan, an experimental treatment for adult patients with platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer who have previously received bevacizumab and mirvetuximab soravtansine.

 

Sofetabart mipitecan is a novel folate receptor alpha (FRα) antibody-drug conjugate (ADC) leveraging proprietary linker technology and an exatecan payload to target tumor cells. Breakthrough Therapy designation is granted to accelerate the development of drugs that treat serious conditions and show preliminary evidence of substantial improvement over existing therapies.

 

“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. 

 

“The Breakthrough Therapy designation and preliminary clinical data for sofetabart mipitecan across all levels of FRα expression are encouraging and point to its potential as a meaningful treatment option for patients.”

 

Jacob Van Naarden, executive vice president and president of Lilly Oncology, added, “We are pleased the FDA has granted Breakthrough Therapy designation for sofetabart mipitecan, reflecting the significant unmet need in platinum-resistant ovarian cancer and the promising initial results shown in our Phase 1 study. Building on compelling results generated to date, 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 based on early Phase 1a/b results, presented at the 2025 ASCO Annual Meeting and updated at the 2025 ESMO Congress. Data showed tumor responses across all dose levels and FRα expression levels, even in patients who progressed on prior mirvetuximab soravtansine. Sofetabart mipitecan also demonstrated a favorable safety profile, with low rates of interstitial lung disease, peripheral neuropathy, alopecia, and no significant ocular toxicity.

 

The drug has now advanced to the Phase 3 FRAmework-01 trial (NCT07213804), evaluating monotherapy in platinum-resistant ovarian cancer and combination therapy with bevacizumab in platinum-sensitive patients. Lilly is conducting the study globally with the European Network for Gynaecological Oncological Trial groups (ENGOT), the GOG Foundation, and the Asia-Pacific Gynecologic Oncology Trials Group (APGOT).