The U.S. Food and Drug Administration on October 14, 2026, expanded the approved use of Anktiva (nogapendekin alfa inbakicept-pmln) to include adults with high-grade papillary-only bladder cancer that has not spread into the bladder muscle and has not responded to BCG (Bacillus Calmette-Guérin) treatment — making it the first FDA-approved therapy specifically for this patient group.
Anktiva, administered together with BCG directly into the bladder, was previously approved for a related but distinct form of non-muscle invasive bladder cancer (NMIBC) called carcinoma in situ (CIS). The expanded approval was granted to Altor BioScience, LLC, according to the FDA press announcement.
A New Regulatory Approach to Bladder Cancer Evidence
The approval marks a departure from the FDA's conventional drug-review framework. The agency based its decision on evidence from the trial that supported Anktiva's earlier approval for CIS patients, together with scientific similarities between CIS and high-grade papillary tumors.
The FDA noted that the two cancer types frequently occur together, can be difficult to distinguish microscopically, and carry a high risk of returning or progressing once BCG treatment fails.
"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."
— R. Angelo de Claro, M.D., Director, FDA Oncology Center of Excellence
What BCG-Unresponsive NMIBC Means
NMIBC refers to bladder cancer that has not grown into the bladder's muscle layer. CIS forms flat patches along the bladder lining, while papillary tumors grow outward from the lining as small, finger-like projections. High-grade cancers — including the papillary-only type now covered by Anktiva — carry a greater risk of spreading than low-grade variants.
BCG is typically the first-line treatment for high-risk NMIBC. It works by activating the immune system inside the bladder. Sometimes, however, the cancer persists or returns. Adding Anktiva gives the immune system another way to fight the cancer, and the combination can help some people whose cancer was not controlled by BCG alone, the FDA said.
Trial Data Behind the Original CIS Approval
The CIS approval supporting today's expanded indication was based on a clinical trial (NCT03022825) evaluating Anktiva in 77 adults, some of whom also had papillary tumors, according to the FDA. Sixty-two percent of participants achieved a complete response — meaning no high-grade cancer was detected during follow-up assessments.
Of those who achieved a complete response, 58 percent maintained it for at least 12 months, and 40 percent maintained it for at least 24 months.
How Anktiva Is Administered
Anktiva is delivered with BCG directly into the bladder through a catheter. The initial course consists of once-weekly administration for six weeks, with additional treatment determined by the cancer's response. Total treatment duration may extend up to 37 months.
The FDA specifies that treatment should be discontinued if the cancer persists after a second six-week course, returns or progresses, or if side effects become unacceptable.
Side Effects and Prescribing Warnings
The most commonly reported side effects included painful or frequent urination, an urgent need to urinate, blood in the urine, urinary tract infections, and muscle or joint pain. Laboratory abnormalities included elevated blood levels of creatinine and potassium. Chills and fever were also reported.
Anktiva's prescribing information carries a warning that delaying surgery to remove all or part of the bladder can allow the cancer to spread to other parts of the body, which the FDA states can be fatal.
FDA Guidance Under Development
To formalize this evidence-bridging approach for future drug developers, the FDA said it is working toward finalizing its draft guidance titled "Bacillus Calmette-Guérin-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment." The guidance is intended to outline how evidence from CIS patient studies may be applied to support approvals for BCG-unresponsive, high-grade papillary-only NMIBC treatments.
By the Numbers
- 77 adults enrolled in the supporting clinical trial (NCT03022825)
- 62% achieved a complete response (no high-grade cancer detected)
- 58% of complete responders maintained that response for at least 12 months
- 40% of complete responders maintained that response for at least 24 months
- 6 weeks — duration of the initial weekly treatment course
- 37 months — maximum possible treatment duration
Why This Matters
Anktiva's expanded approval fills a significant gap for patients with BCG-unresponsive, high-grade papillary-only bladder cancer, a group that previously had no FDA-approved treatment option specifically for their disease type. The FDA's decision to apply trial evidence across closely related cancer forms may also help guide the development of future treatments, according to the FDA. The agency's planned finalization of its guidance document is expected to give drug developers a clearer framework for pursuing similar evidence-bridging approvals.
Photo credit: Main entrance of FDA Building 1, Silver Spring, Maryland — U.S. Food and Drug Administration (public domain)