FDA to review sBLA for nogapendekin alfa inbakicept for BCG-unresponsive papillary NMIBC

The FDA has accepted for review a supplemental biologics license application (sBLA) seeking expanded approval of nogapendekin alfa inbakicept-pmln (Anktiva; NAI) in combination with BCG for patients with BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with papillary disease without carcinoma in situ, ImmunityBio announced in a May 19, 2026 news release.¹

The agency issued a PDUFA target action date of January 6, 2027.

"Today's acceptance of the supplemental BLA represents an important milestone for ImmunityBio and for patients with BCG-unresponsive NMIBC,” said Richard Adcock, President and CEO of ImmunityBio, in the news release.¹ “ANKTIVA is already approved for patients with CIS with or without papillary disease, and this application has the potential to expand access to patients with papillary-only disease, the larger segment of the BCG-unresponsive population.”

Clinical context

The current filing arrives amid broader discussion about trial design and evidence standards in NMIBC. On May 18, 2026, the FDA convened a public workshop titled “Contemporary Issues in Non-Muscle Invasive Bladder Cancer (NMIBC) Trial Design and Interpretation,” which included discussion of biologic overlap between CIS and papillary histologies and implications for regulatory decision-making in single-arm trials.

The FDA stated in correspondence cited by the company, “In support of this expansion, you have submitted the results of QUILT-3.032 Cohort B and a literature-based rationale proposing that papillary NMIBC has an overlapping clinical and non-clinical profile with CIS that may allow for extrapolation of results from patients with CIS, as was demonstrated in QUILT-3.032 Cohort A, the basis of the existing indication, to those with papillary-only disease.”

The agency further noted that “the scientific data detailing these overlapping features will be the focus of the review of this sBLA to determine if there is adequate justification to allow for such an extrapolation and expansion of the indication.”

Adcock added in the news release, “The revelation by the clinicians at the FDA workshop that they treat patients today with papillary disease alone by offering off-label FDA approved therapies for papillary and CIS disease emphasizes the urgent need for this therapy to be made available to patients suffering from high-grade NMIBC. If approved, this expanded indication would further position ANKTIVA plus BCG as an important immunotherapy option across the NMIBC treatment landscape, enabling insurance reimbursement, making this chemotherapy free treatment available to more patients.”

Additional data supporting the sBLA

The application is primarily supported by findings from cohort B of the phase 2/3 QUILT-3.032 study (NCT03022825), which evaluated the regimen in 80 patients with high-grade Ta/T1 papillary-only disease. Results from the trial were published in the Journal of Urology.²

Patients in the study received 400 μg of NAI plus 50 mg intravesical BCG weekly for 6 consecutive weeks.

The study met its primary end point, demonstrating a 12-month disease-free survival (DFS) rate of 58.2% (95% CI, 46.6 to 68.2). At 24 and 36 months, the DFS rates were 52.1% (95% CI, 40.3 to 62.7) and 38.2% (95% CI, 25.6 to 50.6), respectively.

Additional secondary outcomes submitted to the FDA included progression-free survival (PFS), cystectomy-free survival, and disease-specific survival (DSS). The company reported 12-month and 36-month PFS rates of 94.9% and 82.0%, respectively, as well as cystectomy-free survival rates of 92.2% and 83.1% at the same time points. DSS at 36 months was reported as 96.0%, and the median DSS has not yet been reached.

The company also stated that the safety profile observed in cohort B was consistent with prior experience using nogapendekin alfa inbakicept plus BCG and qualitatively similar to BCG alone. The majority (61%) of treatment-related adverse events (TRAEs) were grade 1 to 2. Three percent of patients experienced a grade 3 TRAE, and no grade 4 to 5 TRAEs were reported.

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