FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer

The U.S Food and Drug Administration has expanded the approved use of Anktiva (nogapendekin alfa inbakicept-pmln), given with Bacillus Calmette-Guérin (BCG), to include adults with high-grade papillary-only bladder cancer that has not spread into the bladder muscle and has not responded to BCG treatment. Anktiva is the first FDA-approved treatment specifically for patients with BCG-unresponsive, high-grade papillary-only disease.

The approval marks a new FDA approach to evaluating treatments for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The agency based its decision on evidence supporting an earlier approval of Anktiva for adults with another form of NMIBC, carcinoma in situ (CIS), with or without papillary tumors, and scientific similarities between CIS and high-grade papillary-only disease.

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

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

Treatment for high-risk NMIBC often starts with BCG, which activates the immune system inside the bladder to fight cancer. 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.

Anktiva’s CIS approval was supported by a trial (NCT03022825) that evaluated the treatment’s effectiveness in 77 adults, some of whom also had papillary tumors. Sixty-two percent achieved a complete response, meaning no high-grade cancer was detected during follow-up assessments, and of those, 58% maintained that response for at least 12 months and 40% for at least 24 months.

For today’s expanded approval, the FDA considered these results alongside clinical and laboratory evidence showing that CIS and high-grade papillary tumors share important features. They often occur together, can be difficult to distinguish under a microscope and carry a high risk of returning or progressing when BCG treatment no longer works. These similarities supported applying the evidence of Anktiva’s effectiveness in people with CIS to those with high-grade papillary-only disease.

To help developers apply this approach, the FDA is working toward finalizing its draft guidance, “Bacillus Calmette-Guérin-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment.” The guidance 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.

Anktiva is given with BCG directly into the bladder through a catheter. It is given once a week for six weeks, with additional treatment based on how the cancer responds. Treatment may continue for up to 37 months. It should be stopped if the cancer persists after a second six-week course, returns or progresses, or side effects become unacceptable.

The most common side effects and laboratory abnormalities included painful or frequent urination, an urgent need to urinate, blood in the urine, urinary tract infections, muscle or joint pain, chills, fever, and increased blood levels of creatinine and potassium. 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.

The approval was granted to Altor BioScience, LLC.