Investing.com -- Shares of ImmunityBio, Inc. (NASDAQ: IBRX ) fell 9% following the company’s announcement that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding its supplemental biologics license application (sBLA) for ANKTIVA plus Bacillus Calmette-Guerin (BCG) in the treatment of non-muscle invasive bladder cancer (NMIBC) with papillary disease.
The RTF letter was unexpected, especially considering the positive feedback and unanimous encouragement ImmunityBio received from FDA agency leaders at a January 2025 meeting. The company had been advised to submit the sBLA based on data from a single-arm trial, which showed promising results for patients with NMIBC papillary indication without carcinoma in situ (CIS). Despite this setback, ImmunityBio has already requested an urgent meeting with the FDA to address the inconsistencies between the January guidance and the RTF letter.
ImmunityBio’s ANKTIVA, an interleukin-15 receptor agonist, was previously approved by the FDA in 2024 for use with BCG in treating BCG unresponsive NMIBC with papillary tumors with CIS. The recent RTF letter does not affect this prior approval but has raised concerns within the company and the medical community, given the potential benefits of the treatment for patients with papillary disease without CIS.
Dr. Patrick Soon-Shiong, the Company’s Founder, Executive Chairman, and Global Chief Scientific and Medical Officer, expressed his commitment to continuing the pursuit for approval, emphasizing the strong clinical response and the five-year follow-up data supporting ANKTIVA’s potential. He also highlighted the importance of providing this treatment option to patients faced with the possibility of radical cystectomy and the progression of their disease.
Dr. Rachel Sherman, former FDA Principal Deputy Commissioner, also expressed her surprise at the RTF letter, given the previous consensus at the January meeting. She urged for a rapid resolution to avoid delaying a treatment that could significantly benefit patients at high risk.
The QUILT-3.032 study, which is the basis for the sBLA, is a Phase II/III trial evaluating the efficacy of ANKTIVA in combination with BCG in patients with high-grade NMIBC unresponsive to BCG treatment. The study’s data demonstrated significant disease-specific survival rates and bladder preservation in patients, which ImmunityBio hoped would support the regulatory review and approval process.
ImmunityBio is seeking to clarify the FDA’s decision and expedite the review process for ANKTIVA in the NMIBC papillary indication without CIS. The company remains focused on addressing the high risk of BCG unresponsive bladder cancer and preventing disease progression.
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