FDA accepts NDA for gedatolisib, Celcuity’s investigational breast cancer treatment

The FDA has accepted a New Drug Application (NDA) for Celcuity’s gedatolisib in hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2), PIK3CA wild-type advanced breast cancer, according to today’s news release. A Prescription Drug User Fee Act (PDUFA) goal date has been set for July 17, 2026.

The announcement carries particular importance for breast cancer care because HR+/HER2- disease is the most common breast cancer subtype, accounting for approximately two-thirds of all cases. While advances such as endocrine therapies and CDK4/6 inhibitors have improved outcomes, many patients eventually develop resistance, leading to disease progression and limited treatment options in later lines of therapy. This challenge is especially pronounced for patients with PIK3CA wild-type tumors.

A “wild-type” gene refers to a gene in its natural, non-mutated form. Mutated forms of certain genes, including PIK3CA, have been identified in some cancers and are often the targets of precision therapies. Knowing whether a patient’s tumor harbors a wild-type or mutated gene can be critical in planning cancer treatment, as many targeted drugs are approved only for tumors with specific mutations. Approximately 60% of patients with HR+/HER2- advanced breast cancer have PIK3CA wild-type disease, meaning they lack the mutation targeted by currently approved PI3K inhibitors, leaving a substantial unmet medical need.

The NDA for gedatolisib was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which enables earlier review of key efficacy and safety data before the full application is complete. This is designed to improve efficiency and facilitate faster access to promising oncology treatments without altering the FDA’s standards for approval. Gedatolisib has also previously received Breakthrough Therapy and Fast Track designations, reflecting encouraging early clinical data and the potential to provide meaningful benefit over existing therapies.

The submission is supported by data from the PIK3CA wild-type cohort of Celcuity’s Phase 3 VIKTORIA-1 clinical trial. This trial evaluated gedatolisib in combination with fulvestrant, with or without palbociclib, in patients with HR+/HER2- advanced breast cancer. Results from the wild-type cohort demonstrated clinically meaningful activity, supporting the drug’s potential to fill a therapeutic gap for patients who currently have few targeted options beyond chemotherapy after endocrine resistance.

Gedatolisib is an investigational, multi-target inhibitor of the PI3K/AKT/mTOR (PAM) signaling pathway, a central driver of tumor growth, survival and treatment resistance in breast cancer. Unlike approved therapies that inhibit only a single component of the pathway, gedatolisib simultaneously targets all four Class I PI3K isoforms as well as both mTOR complexes, mTORC1 and mTORC2. This comprehensive blockade is designed to reduce compensatory signaling, or “cross-activation,” that can limit the effectiveness of single-target inhibitors.

In nonclinical studies and early clinical data, gedatolisib has shown comparable potency and cytotoxic activity in both PIK3CA-mutant and PIK3CA wild-type breast cancer cells. This broad activity distinguishes the drug from existing PI3K-pathway therapies and underpins its potential relevance across a larger portion of the HR+/HER2- breast cancer population.

“The FDA’s acceptance of our New Drug Application for gedatolisib and the assignment of a PDUFA goal date is a pivotal milestone in our efforts to bring a much-needed new treatment option to patients with HR+/HER2- advanced breast cancer,” Brian Sullivan, CEO and co-founder of Celcuity, said in the news release. “We believe the robust clinical dataset underlying this submission demonstrates the practice-changing potential of gedatolisib. We are looking forward to collaborating with the FDA throughout the review process as we work towards a potential approval and commercial launch.”