FDA expands Anktiva approval for BCG-unresponsive bladder cancer

The U.S. Food and Drug Administration has approved nogapendekin alfa inbakicept-pmln, marketed as Anktiva, for use with Bacillus Calmette-Guérin (BCG) in adults with BCG-unresponsive, high-grade papillary-only non-muscle invasive bladder cancer (NMIBC). The approval, announced on October 14, 2026, provides the first FDA-approved treatment specifically for this high-risk form of the disease. The drug is administered with BCG directly into the bladder via a catheter once weekly for six weeks, with additional treatment courses based on response, for up to 37 months.

Anktiva's expanded indication rests on evidence extrapolated from its prior approval for carcinoma in situ (CIS). The FDA based its decision on the scientific similarities between CIS and high-grade papillary tumors, as well as data from the earlier approval. The drug was first approved in April 2024 for BCG-unresponsive NMIBC with CIS, with or without papillary tumors. The approval was granted to Altor BioScience, LLC, with the drug marketed by ImmunityBio Inc.

Approval based on prior CIS data and tumor similarities

The FDA expanded the approval using evidence from Anktiva’s prior approval in carcinoma in situ and scientific similarities between CIS and high-grade papillary tumors. This marks a new FDA approach to evaluating treatments for BCG-unresponsive NMIBC. CIS forms flat patches along the bladder lining and is always high-grade. Papillary tumors grow from the lining as small finger-like projections and can be low- or high-grade.

These two tumor types share important features. They frequently occur together and can be difficult to distinguish under a microscope. Both carry a high risk of recurrence or progression after BCG treatment fails. High-grade cancers are more likely to grow and spread than low-grade cancers. These similarities supported applying the evidence of Anktiva’s effectiveness in people with CIS to those with high-grade papillary-only disease.

R. Angelo de Claro, M.D., Director of the FDA’s Oncology Center of Excellence, said, "We listen to patients, clinicians, and researchers, and we act when the science supports a new approach."

The application included results from cohort B of the QUILT-3.032 trial, a phase 2/3, open-label, single-arm, multicenter study. Cohort B enrolled 80 patients with high-grade papillary-only NMIBC. It met its primary endpoint with a 12-month disease-free survival rate of 58.2%. The FDA extended the evidence from the CIS approval to high-grade papillary-only disease without requiring a new trial.

Treatment details and patient outcomes

Anktiva is given with BCG via weekly bladder instillation for six weeks, with additional courses based on response, and has shown durable responses in clinical cohorts. NMIBC refers to bladder cancer that has not grown into the muscle layer of the bladder wall. Treatment for high-risk NMIBC often starts with BCG, which activates the immune system inside the bladder. Adding Anktiva gives the immune system another way to fight the cancer.

The combination can help some people whose cancer was not controlled by BCG alone. Anktiva’s CIS approval was supported by a trial that evaluated the treatment’s effectiveness in 77 adults. Sixty-two percent achieved a complete response. Among those, approximately 58% maintained it for at least 12 months and 40% for at least 24 months.

Depending on response, additional induction and maintenance treatment may be given. Treatment should be stopped if cancer persists after a second six-week course, recurs, progresses, or if adverse effects become unacceptable. The prescribing information warns that delaying bladder removal can allow cancer spread, which can be fatal. The most common adverse effects include painful or frequent urination, urinary urgency, blood in urine, urinary tract infections, muscle or joint pain, chills, fever, and raised blood creatinine and potassium.

FDA advances guidance for future BCG-unresponsive bladder cancer treatments

The FDA is working toward finalizing draft guidance that will support using CIS data to approve treatments for BCG-unresponsive papillary-only disease. The draft guidance is titled 'Bacillus Calmette-Guérin-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment.' It is intended to include discussion of using evidence from studies in patients with CIS to support approval of treatments for BCG-unresponsive, high-grade papillary-only NMIBC.

The FDA is working toward finalizing its draft guidance on using CIS data to support approvals for BCG-unresponsive high-grade papillary-only NMIBC.