FDA Expands Anktiva Approval for Bladder Cancer Patients

The Food and Drug Administration has expanded the Anktiva bladder cancer approval 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 decision, announced on October 14, makes Anktiva, given with Bacillus Calmette-Guerin, known as BCG, the first FDA-approved treatment specifically for patients with this form of the disease. The approval was granted to Altor BioScience, and it arrived ahead of a target date in January 2027 that had been set when the application was accepted.

Anktiva was already approved in April 2024 for a closely related group: adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, with or without papillary tumors. Patients with high-grade papillary tumors alone were not covered by that earlier label, leaving a gap for people whose cancer had returned or persisted after BCG. The new decision closes that gap by extending the same combination to all patients with BCG-unresponsive non-muscle invasive bladder cancer, regardless of whether carcinoma in situ is present, provided the disease is high grade.

The evidence behind the Anktiva bladder cancer approval

Rather than relying on a new trial in the papillary-only group, the FDA based its decision on evidence extrapolated from the earlier trial that supported the carcinoma in situ approval. That trial, Cohort A of QUILT-3.032, evaluated the treatment in 77 adults with BCG-unresponsive, high-risk disease, some of whom also had papillary tumors. In that group, 62 percent achieved a complete response. The agency said the new approach reflects a fresh way of evaluating treatments across closely related forms of bladder cancer, and it is working to finalize guidance on drug development for this condition.

Treatment is delivered through repeated bladder instillations over up to 37 months, with monitoring required, and the prescribing information cautions that delaying surgery to remove the bladder can allow cancer to spread. Patients should discuss the balance of benefits, monitoring, and surgical options with their urology and oncology teams. The expansion does not prove a new response rate for papillary-only disease, but it gives clinicians and patients a first approved option where none existed before.

Source: FDA