FDA Accepts Gedatolisib Application for Some With Breast Cancer

The U.S. Food and Drug Administration (FDA) has accepted a new drug application for gedatolisib in hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-), PIK3CA wild-type advanced breast cancer.

Celcuity Inc. announced in a news release that the regulatory agency granted priority review for the application and assigned a Prescription Drug User Fee Act (PDUFA) goal date of July 17, 2026. This regulatory milestone moves the investigational therapy closer to potential approval for patients who have limited options for managing this specific subtype of advanced disease.

Main data that support the findings

The submission is supported by clinical data from the Phase 3 VIKTORIA-1 clinical trial, specifically focusing on the cohort of patients with PIK3CA wild-type tumors. According to the company, the clinical dataset underlying the application demonstrates that gedatolisib has practice-changing potential for the HR+/HER2- advanced breast cancer population. The mechanism of action for gedatolisib is highly differentiated from currently approved single-target inhibitors that focus on the PI3K/AKT/mTOR (PAM) pathway.

By targeting all four class 1 PI3K isoforms as well as mTORC1 and mTORC2, gedatolisib is designed to induce a comprehensive blockade of the PAM pathway. Scientific data indicates that inhibiting only a single component of this pathway often results in the cross-activation of other components that were not targeted, which can limit the overall suppression of the disease. Gedatolisib is intended to minimize this adaptive cross-activation to enable full suppression of the pathway. Nonclinical studies and early clinical data have shown that the drug maintains comparable potency and cytotoxicity in both PIK3CA-mutant and wild-type breast tumor cells, which distinguishes it from single-target inhibitors.

VIKTORIA-1 Phase 3 trial details

The VIKTORIA-1 Phase 3 trial provided the foundational data for this application, specifically evaluating gedatolisib in patients whose tumors do not have PIK3CA mutations. The new drug application was submitted under the FDA’s Real-Time Oncology Review (RTOR) program. This specific program is utilized by the FDA to facilitate a more efficient and shorter regulatory review period for oncology drugs that may offer significant improvements over existing treatments.

Gedatolisib is categorized as a multi-target PAM inhibitor. While single-target inhibitors have been used in clinical settings, the data from the VIKTORIA-1 trial suggests that the multi-target approach of gedatolisib may address the limitations of those therapies by preventing the pathway from finding alternative ways to remain active. The company stated that the acceptance of the filing and the assignment of the July 17 goal date represents a pivotal milestone in the effort to bring a new treatment option to the patient community.

Safety of the therapy

Regarding safety and the mechanism of the therapy, gedatolisib functions as an investigational, multi-target PI3K/AKT/mTOR inhibitor. It is designed to potently target the various isoforms and complexes within the PAM pathway — specifically all four class 1 PI3K isoforms, mTORC1 and mTORC2 — to ensure the blockade is comprehensive. This comprehensive inhibition is intended to prevent the tumor cells from adapting to the treatment, a common issue seen with single-target inhibitors of the PAM pathway.

The FDA’s decision to grant priority review indicates that the agency will direct intensified attention to the application, as the drug has the potential to provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition. Celcuity Inc. plans to continue collaborating with the FDA throughout the review process as the agency evaluates the clinical data to determine if gedatolisib will be approved for commercial launch in the United States for patients with HR+/HER2- PIK3CA wild-type advanced breast cancer.

Reference

1. “Celcuity Announces FDA Acceptance of New Drug Application for Gedatolisib in HR+/HER2-/PIK3CA Wild-Type Advanced Breast Cancer,” news release; https://ir.celcuity.com/news-releases/news-release-details/celcuity-announces-fda-acceptance-new-drug-application

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI and reviewed by a human editor.

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.