- ImmunityBio (IBRX, Financial) has requested an urgent meeting with the FDA after receiving a Refusal to File (RTF) letter for their supplemental biologics license application (sBLA).
- The sBLA relates to the use of ANKTIVA plus BCG for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) papillary disease.
- This RTF has occurred despite prior unanimous support from FDA leadership to submit the application based on study results.
ImmunityBio, Inc. (IBRX) has sought an urgent meeting with the U.S. Food and Drug Administration (FDA) following a Refusal to File (RTF) letter received for its supplemental biologics license application (sBLA). The application was for the use of ANKTIVA in combination with Bacillus Calmette-Guerin (BCG) to treat BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with papillary disease.
The company submitted the sBLA in March 2025 after receiving clear guidance and encouragement from the FDA in a January meeting, which included representatives from the Center for Biologics Evaluation and Research (CBER), Center for Drug Evaluation and Research (CDER), and the Oncology Center of Excellence (OCE). The FDA had reportedly supported an expedited submission based on positive outcomes from the QUILT 3.032 study.
ANKTIVA had already been approved in 2024 for BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary disease. In presenting the data, ImmunityBio highlighted its 99% disease-specific survival rate at 12 months and an over 82% bladder-sparing rate. These results, considered 'best in class' by experts at the 2025 American Urological Association meeting, underscored the therapy's safety and efficacy.
Dr. Patrick Soon-Shiong, ImmunityBio's Founder, expressed determination to work towards a resolution with the FDA through a requested Type A meeting. The inconsistencies were particularly surprising given the identical protocols and patient populations involved in the study that supported the previously approved indication for papillary disease with CIS.
The company strongly urges the FDA to reconsider, emphasizing the potential life-saving impact for patients who otherwise might face radical cystectomy or disease progression. ImmunityBio is poised to engage in additional FDA discussions to explore the best path forward, potentially including an Advisory Committee meeting.
