Oncology News Central (ONC)FDA Must Explain “Confounding” Bladder Cancer Drug Rejection, Company Says

ImmunityBio received an FDA Refusal to File letter for expanding Anktiva plus BCG indication to BCG-unresponsive non-muscle invasive bladder cancer patients with papillary disease without carcinoma in situ, despite previous agency encouragement and existing approval for similar patient populations.

⚕️ Key Clinical Considerations ⚕️

• Regulatory Inconsistency: FDA approved Anktiva plus BCG for papillary tumors with CIS but refused filing for papillary without CIS, despite both populations being studied in the same QUILT-3.032 trial with identical treatment protocols.
• Clinical Efficacy Data: Phase 2/3 trial demonstrated 55.4% disease-free survival at 12 months, 48.3% at 24 months, with median DFS of 19.3 months in 80 BCG-unresponsive patients with high-grade Ta/T1 papillary disease.
• Safety Profile: Treatment showed excellent tolerability with only 7% of patients requiring cystectomy at median 20.7-month follow-up, supporting the drug’s bladder-sparing potential in this high-risk population.
• Expert Criticism: Former FDA official Rachel Sherman characterized the refusal as containing “regulatory inaccuracies” and contradicting previous agency guidance, highlighting potential procedural concerns in the review process.
• Patient Impact: Denial affects access to potential cystectomy-avoiding treatment for BCG-unresponsive NMIBC patients, a population with limited therapeutic options and significant quality-of-life implications from radical surgery.

🎯 Clinical Practice Impact 🎯

• Patient Communication: Discuss treatment uncertainty for BCG-unresponsive papillary NMIBC patients while regulatory issues resolve, emphasizing continued monitoring and alternative management strategies.
• Practice Integration: Monitor FDA resolution timeline and prepare for potential approval reversal, ensuring treatment protocols accommodate possible future access to combination therapy.
• Risk Management: Counsel patients on cystectomy timing versus waiting for regulatory clarification, balancing disease progression risk against potential access to bladder-sparing treatment options.
• Action Items: Track regulatory updates, review current BCG-unresponsive patient populations for potential future treatment candidates, and maintain documentation supporting treatment decisions during regulatory uncertainty.

More on Bladder Cancer