FDA Expands Anktiva Approval in BCG-Unresponsive Bladder Cancer

The Food and Drug Administration (FDA) has expanded the approval of Anktiva (nogapendekin alfa inbakicept-pmln) given with Bacillus Calmette-Guérin (BCG) to include adults with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) whose tumors are high-grade and papillary-only.

Anktiva with BCG was previously approved for adults with BCG-unresponsive NMIBC with carcinoma in situ (CIS), with or without papillary tumors. The Oct. 14 decision broadens that indication so patients whose cancer did not respond to BCG and whose tumors are papillary without CIS can also receive the treatment.

Why did the FDA expand the Anktiva approval in bladder cancer?

Rather than relying on a new trial, the FDA based the expanded approval on a review of available scientific information. High-grade papillary tumors and CIS grow differently, but the agency noted that both are considered aggressive forms of urothelial carcinoma, a cancer that begins in the cells lining the urinary tract. The two tumor types often appear together and can be difficult to tell apart under a microscope. In patients whose cancer has not responded to BCG, both also carry a high risk of coming back or progressing.

Because of these overlapping features, the FDA concluded that the benefit previously shown in patients with CIS could be extended to patients with high-grade papillary disease.

What did the QUILT-3.032 trial show for Anktiva in BCG-unresponsive bladder cancer?

The efficacy data behind Anktiva come from Cohort A of the QUILT-3.032 trial, which enrolled 77 patients with BCG-unresponsive, high-risk NMIBC with CIS, with or without papillary tumors. The trial’s main measure of whether the treatment worked was complete response, meaning cystoscopy (a camera exam of the bladder), urine cytology (a urine test that looks for cancer cells) and biopsy showed no signs of cancer.

In the trial, 62% of patients had a complete response. Duration of response, or how long a response lasted, was an additional measure. Among patients who had a complete response, 58% had a response lasting 12 months or longer and 40% had a response lasting 24 months or longer.

QUILT-3.032 was a single-arm trial conducted at multiple centers, meaning every patient received Anktiva with BCG and there was no comparison group. Patients entered the trial after transurethral resection, a procedure that removes bladder tumors through the urethra. They then received Anktiva with BCG placed directly into the bladder, followed by maintenance treatment for up to 37 months.

What side effects were reported with Anktiva?

The most common side effects, reported in at least 15% of patients and including changes seen in lab tests, were increased creatinine (a marker of kidney function), painful urination, blood in the urine, frequent urination, an urgent need to urinate, urinary tract infection, increased potassium, muscle and bone pain, chills and fever.

The prescribing information also warns that delaying cystectomy, or surgery to remove the bladder, can lead to bladder cancer that spreads to other parts of the body, which can be lethal.

How is Anktiva given for bladder cancer?

Anktiva is given at a dose of 400 mcg into the bladder with BCG once a week for six weeks as the first, or induction, phase of treatment. Patients who still have CIS, high-grade Ta tumors or both at month 3 may receive a second induction course.

Maintenance treatment follows, with 400 mcg given with BCG once a week for three weeks at months 4, 7, 10, 13 and 19, for a total of 15 doses. Patients with no evidence of high-grade disease at month 25 and later may continue maintenance with BCG once a week for three weeks at months 25, 31 and 37, for up to nine additional instillations.

Treatment is stopped if cancer remains after a second induction course, if it comes back or progresses, or if side effects become too difficult to manage. Treatment lasts no longer than 37 months.

What does the expanded Anktiva approval mean for patients with bladder cancer?

Patients with BCG-unresponsive NMIBC whose tumors are high-grade and papillary-only now have Anktiva with BCG as an approved treatment, which until now required disease that included CIS. The response data supporting the approval were gathered in patients with CIS, and the FDA extended those findings to papillary-only disease based on the features the two tumor types share.

Reference

1. “FDA approves an expanded indication for nogapendekin alfa inbakicept-pmln for BCG-unresponsive non-muscle invasive bladder cancer.” U.S. Food and Drug Administration. Posted: Oct. 14, 2026.

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