ImmunityBio Slapped With FDA Refusal To File Letter For Expanded Use Of Its Bladder Cancer Drug

ImmunityBio, Inc. IBRX on Monday received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for the supplemental biologics license application (sBLA) for use of ANKTIVA plus Bacillus Calmette-Guerin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) for the indication of papillary disease.

This RTF letter was received despite unanimous guidance and encouragement from the agency’s leadership, including from CBER, CDER, and OCE, to submit this sBLA at the in-person January 2025 meeting.

At this meeting, all key decision makers were specifically asked and unanimously confirmed that ImmunityBio should submit the sBLA as soon as possible based on the data in the single-arm trial.

Also Read: ImmunityBio Analyst Predicts Strong 2025 For New Bladder Cancer Drug

Relying on this unanimous guidance, the company submitted the sBLA in March 2025. The company has already requested an urgent meeting to resolve the inconsistencies between the directives provided at the January Meeting and receipt of the RTF letter.

ANKTIVA was approved by the FDA in 2024 with BCG for BCG unresponsive NMIBC with Papillary tumors with CIS.

In the same clinical trial (QUILT-3.032), the long-term results of patients with Papillary tumors without CIS (Cohort B) were submitted as an sBLA. The RTF letter referenced herein does not impact this prior approval of CIS +/—papillary in BCG-unresponsive patients.

Data was also presented on the long-term follow-up Phase 1 results, which demonstrated Complete Response and Disease-Free Survival in both CIS and Papillary disease, with patients in an ongoing cystectomy-free state at 10 years.

In addition, the disease-specific overall survival rate of 99% at 12 months and 96% at 36 months in the BCG unresponsive patients with Papillary disease without CIS was discussed.

In March 2025, the company completed its submission to the FDA of the sBLA for the use of ANKTIVA plus BCG in BCG-unresponsive NMIBC in the papillary indication.

The company and its consultants, who attended the January 2025 meeting, were shocked by this inconsistent response and have requested an urgent meeting with the FDA to resolve this issue.

In April last year, ImmunityBio announced the overall survival results of Anktiva combined with checkpoint inhibitors in non-small cell lung cancer.

QUILT 3.055 trial completed and shows median overall survival almost double that of standard of care chemotherapy in 2nd- and 3rd-line non-small cell lung cancer (NSCLC) patients whose cancer did not respond to checkpoint inhibitors with or without chemotherapy.

Price Action: At the last check on Monday, IBRX stock was down 16.50% to $2.12 during the premarket session.

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