FDA Approves First Treatment for BCG-Unresponsive Bladder Cancer

The Food and Drug Administration 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. The agency said 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. The agency based its decision on evidence supporting an earlier approval of Anktiva for adults with carcinoma in situ (CIS), with or without papillary tumors, and scientific similarities between CIS and high-grade papillary-only disease.

The approval was supported by a trial (NCT03022825) that evaluated the treatment in 77 adults. Sixty-two percent achieved a complete response, meaning no high-grade cancer was detected during follow-up assessments. Of those, 58 percent maintained that response for at least 12 months and 40 percent for at least 24 months.

The agency said 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.

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.

The most common side effects 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. The agency warned 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.