The U.S. Food and Drug Administration (FDA) has expanded the approved use of the drug 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 treatment. This approval marks the first time the FDA has specifically approved a treatment for patients with BCG-unresponsive, high-grade papillary-only disease.
Anktiva is administered directly into the bladder through a catheter in combination with Bacillus Calmette-Guérin (BCG). The treatment is given once a week for six weeks, with additional treatment based on patient response. The regimen may continue for up to 37 months, though it should be stopped if the cancer persists after a second six-week course, returns, progresses, or if side effects become unacceptable.
The agency based this decision on evidence supporting an earlier approval of Anktiva for adults with carcinoma in situ (CIS), with or without papillary tumors. The FDA determined that CIS and high-grade papillary-only disease share important features, including a high risk of returning or progressing when BCG treatment is no longer effective. This scientific similarity allowed the agency to apply the results from a clinical trial evaluating Anktiva in patients with CIS to support the new approval.
According to the trial referenced (NCT03022825), which evaluated 77 adults, 62 percent achieved a complete response, meaning no high-grade cancer was detected during follow-up assessments. Of those who responded, 58 percent maintained that response for at least 12 months and 40 percent for at least 24 months.
The most common side effects and laboratory abnormalities associated with the treatment include 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 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. The FDA is working toward finalizing a draft guidance regarding the development of drugs and biological products for BCG-unresponsive nonmuscle invasive bladder cancer, which includes discussion of using evidence from studies in patients with CIS to support approvals for high-grade papillary-only disease.
