FDA widens ImmunityBio's Anktiva approval to papillary-only bladder cancer months ahead of deadline

The Food and Drug Administration on Wednesday expanded ImmunityBio's bladder cancer drug Anktiva to patients whose high-grade papillary tumors no longer respond to standard BCG therapy, acting about 12 weeks before its Jan. 6, 2027 deadline and leaning on evidence from the drug's earlier approval.

What the FDA approved

Anktiva, ImmunityBio's immunotherapy given with the standard bladder cancer treatment Bacillus Calmette-Guérin, or BCG, is now approved for adults with BCG-unresponsive non-muscle invasive bladder cancer, or NMIBC, without the earlier restriction to one tumor type. Since April 2024 the label had covered patients with carcinoma in situ, a flat, always high-grade cancer along the bladder lining, with or without papillary tumors. Wednesday's revision adds patients whose disease is high-grade papillary tumors alone.

The FDA said Anktiva is the first treatment it has approved specifically for that group. The approval was granted to Altor BioScience, an indirect wholly owned subsidiary of ImmunityBio. The decision came about 12 weeks before the Jan. 6, 2027 target action date the agency had set for the application under the Prescription Drug User Fee Act, known as the PDUFA date.

A different way of using the evidence

The FDA's notices do not cite results from papillary-only patients. The agency said it based the decision on the data that supported the 2024 approval and on scientific similarities between the two forms of disease. Those data come from Cohort A of the QUILT-3.032 study, in which 62% of 77 patients with carcinoma in situ had a complete response. Among those responders, 58% kept the response for at least 12 months and 40% for at least 24 months.

The FDA said the two forms of disease often occur together, can be hard to tell apart under a microscope and carry a high risk of coming back or progressing once BCG stops working. On that basis it concluded the earlier efficacy results could be extended to high-grade papillary-only disease, and it called the decision a new approach to evaluating treatments in this setting.

"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," said R. Angelo de Claro, director of the FDA's Oncology Center of Excellence. "This approach may also help guide the development of future treatments." The agency said it is working to finalize draft guidance that will discuss using evidence from carcinoma in situ studies to support approvals in papillary-only disease.

From refusal to early approval

The approval ends a long fight over this indication. In May 2025 the FDA sent ImmunityBio a refusal-to-file letter for its papillary application, declining to review it. The company said at the time the letter contradicted encouragement it had received from agency leadership at a January 2025 meeting. In its August second-quarter report, ImmunityBio said the FDA had accepted a papillary application for review and set the Jan. 6, 2027 target date.

What it means for the business

Anktiva is ImmunityBio's main commercial product. The company reported second-quarter net product revenue of $50.7 million, up 92% from a year earlier, and first-half revenue of $94.8 million. It ended June with $357.4 million in cash, cash equivalents and marketable securities.

ImmunityBio says papillary disease makes up about 85% of the non-muscle invasive bladder cancer population and that the new label significantly increases the drug's U.S. addressable market. It has not put a number on how many BCG-unresponsive papillary-only patients it can now reach or on any revenue effect. "The expansion of ANKTIVA plus BCG into papillary-only disease broadens the eligible patient population," said Chief Executive Richard Adcock.

Investors were measured. IBRX shares traded as high as $10.46 on Wednesday before closing at $9.91, up 1.1% from Tuesday's $9.80, on volume of about 30 million shares versus about 11.5 million the day before, according to StockAnalysis market data.

The competitive picture

A rival intravesical therapy, TAR-200, is being tested in the same papillary-only population through SunRISe-5, a randomized phase 3 study whose primary endpoint is disease-free survival. Its ClinicalTrials.gov record lists Janssen Research & Development, LLC as lead sponsor. The study enrolls patients whose papillary-only disease returned within a year of BCG and who declined or could not have their bladder removed. How the FDA's finalized guidance treats programs like that will shape whether rivals can follow the path Anktiva just took.

Safety and use

Anktiva is given with BCG directly into the bladder through a catheter, weekly for six weeks, with further treatment depending on response for up to 37 months. The most common side effects included painful or frequent urination, blood in the urine, urinary tract infections, chills and fever. The label warns that delaying surgery to remove the bladder can allow the cancer to spread, which can be fatal.