FDA Expands Use of Anktiva to Treat High-Grade Papillary Non‑Muscle Invasive Bladder Cancer

The U.S. Food and Drug Administration on Oct. 14 expanded the approved use of Anktiva to adults with BCG-unresponsive, high-grade papillary-only non-muscle invasive bladder cancer, making it the first FDA-approved treatment specifically for that group of patients. The decision broadens the label for the ImmunityBio drug, rather than marking its first approval.

The move matters because it adds a bladder-preserving option for patients whose cancer has not spread into the bladder muscle but has stopped responding to BCG, a longstanding bladder-cancer treatment. For patients with BCG-unresponsive, high-grade papillary non-muscle invasive disease, options have been limited, and radical cystectomy, or bladder-removal surgery, has often been a standard treatment after BCG failure.

The FDA also cast the decision as a regulatory first. The agency said it based the expansion on evidence from Anktiva’s earlier approval in a related bladder-cancer subtype, BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, or CIS, along with clinical and laboratory evidence showing scientific similarity between CIS and high-grade papillary tumors. “We listen to patients, clinicians, and researchers, and we act when the science supports a new approach. 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,” said Dr. R. Angelo de Claro, director of the FDA’s Oncology Center of Excellence, in the agency’s press release. The FDA said it is working to finalize draft guidance that would discuss using CIS study evidence to support approvals for papillary-only disease.

Anktiva, known generically as nogapendekin alfa inbakicept-pmln, was first approved on April 22, 2024, for use with BCG in adults with BCG-unresponsive non-muscle invasive bladder cancer with CIS, with or without papillary tumors. In the trial supporting that earlier approval, the FDA said the complete response rate was 62%. Among patients who responded, 58% maintained that response for at least 12 months and 40% for at least 24 months.

The pivotal development program was QUILT-3.032, a multicenter, open-label phase 2/3 trial listed on ClinicalTrials.gov as NCT03022825. The evidence base came from a single-arm study, meaning patients were not compared with a randomized control group.

Anktiva was approved for the expanded use for Altor BioScience LLC, an indirect wholly owned subsidiary of ImmunityBio Inc. The treatment is given intravesically, meaning directly into the bladder through a catheter, with BCG once weekly for six weeks as an initial course. Additional treatment depends on how the patient responds and may continue for up to 37 months. Treatment should be stopped if the cancer persists after a second six-week course, recurs or progresses, or side effects become unacceptable. The FDA said Anktiva’s prescribing information warns that delaying surgery to remove all or part of the bladder can allow the cancer to spread to other parts of the body, which can be fatal. Common side effects and laboratory abnormalities listed by the agency include painful or frequent urination, urgency, blood in the urine, urinary tract infection, muscle and joint pain, chills, fever, and increased creatinine and potassium.