The US Food and Drug Administration (FDA) has expanded the approved use of Anktiva (nogapendekin alfa inbakicept-pmln), in combination with Bacillus Calmette-Guérin (BCG), to include adults with high-grade papillary-only non-muscle-invasive bladder cancer that has not responded to BCG treatment.
The expanded approval, announced on October 14, 2026, provides a new treatment option for patients with this form of bladder cancer. According to the FDA, Anktiva is the first specifically approved treatment for adults with BCG-unresponsive, high-grade papillary-only non-muscle-invasive bladder cancer. The approval was granted to Altor BioScience, LLC, an indirect wholly owned subsidiary of ImmunityBio, Inc.
Before this decision, Anktiva was approved in combination with BCG for adults with BCG-unresponsive non-muscle-invasive bladder cancer (NMIBC) involving carcinoma in situ (CIS), with or without papillary tumours. The expanded indication now includes patients with high-grade papillary-only tumours, even when carcinoma in situ is not present.
Non-muscle-invasive bladder cancer refers to cancer that has not grown into the muscular layer of the bladder wall. It includes different forms of disease, including carcinoma in situ and papillary tumours.
Carcinoma in situ consists of flat cancerous lesions along the bladder lining, whereas papillary tumours grow outward from the lining in finger-like projections. High-grade papillary tumours are associated with a greater risk of recurrence and progression than low-grade tumours.
BCG is an established treatment for high-risk non-muscle-invasive bladder cancer. However, some patients experience persistent or recurrent disease despite receiving BCG. The expanded approval addresses this unmet treatment need. The FDA based its decision on a review of available scientific evidence rather than a new clinical trial specifically conducted in patients with papillary-only disease. The agency considered evidence from the earlier Anktiva approval, alongside clinical and non-clinical similarities between carcinoma in situ and high-grade papillary tumours.
According to the FDA, these forms of bladder cancer can occur together, may be difficult to distinguish pathologically and share important features associated with aggressive disease. Both can carry substantial risks of recurrence and progression when the cancer no longer responds to BCG.
Based on these similarities, the FDA concluded that the previously demonstrated efficacy of Anktiva in patients with carcinoma in situ could be extrapolated to patients with BCG-unresponsive high-grade papillary-only disease.
The decision also signals a regulatory approach in which evidence from one closely related form of cancer may support an expanded indication when scientific similarities justify that conclusion.
The original approval of Anktiva was supported by Cohort A of the Phase 2/3 QUILT-3.032 clinical trial, which included 77 patients with BCG-unresponsive, high-risk non-muscle-invasive bladder cancer involving carcinoma in situ, with or without papillary disease.
Patients received Anktiva through intravesical administration in combination with BCG, followed by maintenance treatment for up to 37 months.
The FDA reported the following outcomes:
- 62% complete response rate: The proportion of patients who achieved a complete response based on assessments including cystoscopy, urine cytology and biopsy.
- 58% sustained response: Among patients who achieved a complete response, 58% maintained the response for at least 12 months.
- 40% longer-term response: Among complete responders, 40% maintained the response for at least 24 months.
These results supported the original indication. The FDA used the totality of the available scientific evidence to justify extending the indication to high-grade papillary-only disease.
Importantly, the reported response figures are from the original study population and should not be interpreted as response rates established specifically in patients with papillary-only tumours.
Anktiva is administered directly into the bladder through a catheter in combination with BCG. According to the FDA's recommended regimen, the dose is 400 micrograms once weekly for six weeks during induction treatment. A second induction course may be administered in specified circumstances if residual carcinoma in situ and/or high-grade Ta tumours remain at month three.
Maintenance treatment consists of weekly administration for three weeks at months 4, 7, 10, 13 and 19. For eligible patients without evidence of high-grade disease at month 25 and beyond, additional maintenance courses may be given at months 25, 31 and 37.
Treatment should be discontinued if disease persists after a second induction course, recurs or progresses, or if toxicity becomes unacceptable. The maximum treatment duration is 37 months. Treatment decisions and administration must follow the approved prescribing information and be supervised by qualified healthcare professionals. The expanded indication increases the treatment options available to adults whose high-grade papillary-only bladder cancer does not respond to BCG.
It also demonstrates how regulatory authorities may use established clinical evidence and biological similarities between disease subtypes to support an expanded indication without requiring a separate efficacy trial in every closely related patient population.
The FDA is working toward finalising guidance on developing medicines for BCG-unresponsive non-muscle-invasive bladder cancer, including the use of evidence from patients with carcinoma in situ to support treatment development for high-grade papillary-only disease.
The decision represents an important regulatory development in bladder cancer treatment, although the benefits and risks of Anktiva must be evaluated for each patient.
