FDA Approves First Treatment for BCG-Unresponsive Bladder Cancer

Nogapendekin alfa inbakicept-pmln (Anktiva; ImmunityBio Inc) given with Bacillus Calmette-Guérin (BCG) is now the first FDA-approved treatment specifically for adults with BCG-unresponsive, high-grade papillary-only nonmuscle invasive bladder cancer (NMIBC), according to an FDA announcement.¹

The expanded indication covers adults whose high-grade papillary-only disease has not spread into the bladder muscle and has not responded to BCG. The agency based its decision on the evidence that supported nogapendekin alfa inbakicept-pmln earlier approval for BCG-unresponsive carcinoma in situ (CIS), with or without papillary tumors, combined with the scientific similarities between the 2 forms of disease. The approval was granted to Altor BioScience, LLC.

“We listen to patients, clinicians, and researchers, and we act when the science supports a new approach,” said R. Angelo de Claro, MD, director of the FDA’s Oncology Center of Excellence, in a statement. “For Anktiva, our review of the similarities between these cancers allowed us to build on the results in patients with CIS and expand treatment options for people with high-grade papillary tumors alone. This approach may also help guide the development of future treatments.”

Extrapolating CIS Results to Papillary-Only Disease

NMIBC has not grown into the bladder's muscle layer and can include both CIS and papillary tumors. CIS forms flat patches along the bladder lining and is always high-grade, whereas papillary tumors grow from the lining as small, finger-like projections and can be low- or high-grade. High-grade cancers are more likely to grow and spread.

The CIS approval was supported by a trial (NCT03022825) of 77 adults, some of whom also had papillary tumors. In that trial, 62% achieved a complete response, defined as no high-grade cancer detected during follow-up. Among responders, 58% maintained the response for at least 12 months and 40% for at least 24 months.

For the expansion, the FDA weighed those results alongside clinical and laboratory evidence that CIS and high-grade papillary tumors share important features. The 2 frequently occur together, can be difficult to distinguish under a microscope, and carry a high risk of recurrence or progression once BCG stops working. The agency said it is working toward finalizing draft guidance on developing drugs for BCG-unresponsive NMIBC that is intended to address using CIS data to support approvals in papillary-only disease.

Approval Arrives Ahead of January 2027 Target Date

ImmunityBio, which markets nogapendekin alfa inbakicept-pmln, announced in May 2026 that the FDA had accepted its supplemental biologics license application for papillary disease without CIS and assigned a Prescription Drug User Fee Act target date of January 6, 2027.² According to the company, the FDA's filing communication said the review would focus on whether data on the overlapping features of papillary disease and CIS justified extending the indication, while reiterating the agency's concerns about single-arm trials in papillary disease alone.

The application included results from cohort B of QUILT-3.032, which enrolled 80 patients with high-grade papillary-only NMIBC. The company reported that the cohort met its primary end point, with a 12-month disease-free survival rate of 58.2% (95% CI, 46.6%-68.2%). Nogapendekin alfa inbakicept-pmln plus BCG was first approved in April 2024 for BCG-unresponsive NMIBC with CIS, with or without papillary tumors.

Dosing and Safety

Nogapendekin alfa inbakicept-pmln is administered with BCG directly into the bladder through a catheter once weekly for 6 weeks, with further treatment based on response, for up to 37 months.¹ Treatment should be stopped if cancer persists after a second 6-week course, recurs, or progresses, or if adverse effects become unacceptable.

The most common adverse effects and laboratory abnormalities included painful or frequent urination, urinary urgency, blood in the urine, urinary tract infections, muscle or joint pain, chills, fever, and elevated blood creatinine and potassium levels. The prescribing information warns that delaying partial or complete bladder removal can allow the cancer to spread, which can be fatal.

References

1. FDA approves first treatment for certain people with BCG-unresponsive bladder cancer. News release. FDA. October 14, 2026. Accessed October 14, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-certain-people-bcg-unresponsive-bladder-cancer
2. ImmunityBio announces FDA acceptance of supplemental BLA for Anktiva plus BCG in BCG-unresponsive non-muscle invasive bladder cancer with papillary disease; PDUFA date set for January 6, 2027. News release. ImmunityBio. May 19, 2026. Accessed October 14, 2026. https://ir.immunitybio.com/news-releases/news-release-details/immunitybio-announces-fda-acceptance-supplemental-bla-anktiva