On January 19, Patrick Soon-Shiong went on The Sean Spicer Show for an episode titled “Is the FDA BLOCKING Life Saving Cancer Treatments?” and told listeners that Anktiva, the drug his company ImmunityBio sells, was “approved for bladder cancer, but it actually can treat all cancers.” The grievance was in the episode title. Eight months earlier, the agency had refused even to open ImmunityBio’s application to sell Anktiva to a larger group of bladder cancer patients.
cannot be meaningfully interpreted without an appropriate comparator
On October 14 the FDA gave him that label. The agency announced that Anktiva (nogapendekin alfa inbakicept-pmln), given with BCG, is now approved for adults whose high-grade, papillary-only non-muscle invasive bladder cancer no longer responds to BCG, and it called the drug the first treatment for that group. The decision came twelve weeks ahead of the agency’s own January 6, 2027 deadline. As its evidence, the announcement pointed to 77 patients from ImmunityBio’s pivotal trial who had carcinoma in situ, “some of whom also had papillary tumors.” That same trial had also enrolled 80 patients with papillary-only disease, which is precisely the population on the new label. The announcement cited the 77. It did not cite the 80.
Between the podcast and the approval, the FDA’s Office of Prescription Drug Promotion sent ImmunityBio a March 13 warning letter that quoted the podcast back to the company, including the line “We have the therapy to prevent cancer if you were exposed to radiation.” According to Covington’s summary of the letter, the agency called the cancer-vaccine and prevention claims false and said the company had failed to file the podcast with the FDA when it first aired, as regulations require. It was the third letter since September to ImmunityBio or its subsidiary over how the drug was being sold.
Patients with nothing left to shrink
Non-muscle invasive bladder cancer has not yet grown into the bladder’s muscle wall. The standard treatment is BCG, a weakened bacterium instilled into the bladder to provoke an immune attack, and when BCG stops working the usual recommendation is to remove the bladder. Anktiva’s own approval notice warns that delaying that surgery “can lead to the development of metastatic bladder cancer, which can be lethal.” A patient who keeps their bladder on a drug like this is betting against that sentence.
The disease takes two forms. Carcinoma in situ (CIS) is flat and spreads along the bladder lining, while papillary tumors grow outward and are cut out before treatment starts, so a papillary-only patient begins therapy with no visible cancer left. ImmunityBio says that about 85 percent of the 64,000 Americans diagnosed with this kind of bladder cancer each year present with papillary disease, and its chief executive, Richard Adcock, calls the papillary-only group “the larger segment of the BCG-unresponsive population.” The October decision opened the label to that segment.
For regulators the two forms have never been interchangeable, because they produce different kinds of evidence. When CIS is present at the start of a trial, investigators can count complete responses: the disease was visible before treatment and is gone afterward, so even a single-arm study has something to measure. A papillary patient whose tumor has already been removed has nothing left to clear. The only thing a drug can do for that patient is keep the cancer from returning, and recurrence is a time-to-event outcome. Some patients stay free of disease for a long time whatever they receive, which is why a single arm can tell you how many patients stayed well but not how many stayed well because of the drug. The FDA’s 2024 draft guidance says patients without active disease “should be included in RCTs using a time-to-event primary endpoint,” and that resected patients without CIS may enroll in single-arm trials but should be left out of the primary efficacy analysis.
- 2024first approval on the CIS cohort
- 2025papillary filing refused
- 2026papillary-only approval by extrapolation
- 2030rival randomized trial's estimated completion
ImmunityBio’s pivotal trial, QUILT-3.032, ran single-arm cohorts. Cohort A, 77 patients with CIS with or without papillary tumors, earned the April 2024 approval: 62 percent had a complete response, and among those responders 58 percent stayed in response for at least 12 months and 40 percent for at least 24. Cohort B enrolled the 80 patients with papillary-only disease.
To bridge the two, the FDA offered an argument instead of data. It wrote that the two forms “often occur together,” “can be difficult to distinguish under a microscope,” and carry a high risk of recurrence or progression once BCG fails, and on that basis it applied the CIS evidence to the papillary-only population. At an FDA public workshop on May 18, panelists said the two arise from the same cancer-causing clone, according to ImmunityBio’s account of the session. That is a respectable biological case, but it answers a different question from the one a papillary patient asks. It shows that the combination can clear flat disease that is already present. It does not measure how long the combination keeps a removed tumor from coming back.
The numbers nobody leaned on
Cohort B’s results are public, published by the sponsor’s investigators in the Journal of Urology and summarized in the trade press. Of the 80 patients, 58.2 percent were free of disease at 12 months, 52.1 percent at 24 months and 38.2 percent at 36 months, and median disease-free survival was 25.3 months. The bladder figures look better: 92.2 percent had avoided cystectomy at 12 months and 81.8 percent at 36. The cohort was 74 percent male and 89 percent white, and it had no control arm.
| Group | Value (percent) |
|---|---|
| 12 months | 58.2 |
| 24 months | 52.1 |
| 36 months | 38.2 |
The FDA had already told the same company, in writing, what it thought of numbers like these. In a January 7, 2026 untitled letter to ImmunityBio’s subsidiary Altor BioScience, the agency objected to Anktiva websites claiming that 84 percent of responders kept their bladders at 36 months and that disease-specific survival reached 99 percent. Because QUILT-3.032 had no comparator, and because cystectomy avoidance and survival are time-to-event endpoints, the agency wrote that the results “cannot be meaningfully interpreted without an appropriate comparator.” It added that it could not verify whether the outcomes reflected Anktiva or the natural course of the disease. The March warning letter added that the trial’s Anktiva-alone cohort had been stopped early for futility, so whatever efficacy exists belongs to Anktiva plus BCG.
Those letters concerned advertising, not approval, and an agency can consistently bar a company from marketing uncontrolled recurrence figures while approving the drug on response data from a related form of the disease. That is roughly what happened here. It leaves the papillary-only patients on the new label covered by a response rate measured in other people, while the only recurrence numbers from patients like them are ones the FDA has said, in writing, that it cannot interpret.
Seventeen months and the same 77 patients
The regulatory history turned three times. First came the refusal. ImmunityBio says senior FDA officials encouraged it in January 2025 to file for papillary disease on its existing trial data, and on May 2, 2025, the agency issued a refusal-to-file letter. Soon-Shiong argued that the label already covered papillary tumors when they came with CIS, and that both groups had come from the same trial. The FDA published no rationale.
The second turn was a resubmission without a new trial. After a January 2026 meeting the agency asked for additional data and, according to OncLive, “did not ask for the initiation or design of any new clinical trials.” ImmunityBio sent the data in February and announced the resubmission on March 9, four days before the warning letter. That same month NCCN listed the combination as a category 2A option for papillary-only disease, the company reported.
The third turn was approval by extrapolation. When the FDA accepted the filing in May, it said “the scientific data detailing these overlapping features will be the focus of the review,” and, by the company’s account, reiterated its concerns about “single-arm trials in papillary disease alone.” Five months later it approved the drug early.
The agency owes the public an account of what changed. The cohort it cited in October is the one that carried the April 2024 approval, and the overlap biology predates both the refusal and the approval. Between those two decisions came an overlapping-features package the FDA itself requested, a public workshop and longer follow-up. Any of those could have moved the reviewers, but the announcement does not say which did, and the review documents that would show how hard anyone pushed on the extrapolation were not released with it. In the meantime, patients who had run out of BCG spent seventeen months making decisions about their bladders without an FDA label to guide them.
Who collects
Anktiva’s wholesale list price was $35,800 per 400-microgram vial in the company’s June 2025 disclosure, a figure that excludes discounts and rebates. The label allows six weekly induction doses, 15 maintenance doses through month 19 and up to nine more through month 37 for patients still free of high-grade disease. A patient who stays well through the full schedule receives 30 instillations, roughly $1.07 million at the 2025 list price, before the BCG is counted.
ImmunityBio reported second-quarter 2026 net product revenue of $50.7 million, up 92 percent from a year earlier and earned while CIS was the drug’s only approved indication. Every Anktiva dose is given alongside BCG, and in the same filing the company disclosed that it had secured exclusive U.S. rights to develop, import and commercialize Tokyo-172 BCG. ImmunityBio now has rights to both halves of the combination the label requires.
The randomized design the draft guidance describes is not hypothetical. Johnson & Johnson’s SunRISe-5 enrolled 272 patients whose papillary-only, high-risk disease came back within a year of BCG and randomized them to its TAR-200 device or to standard intravesical chemotherapy, with disease-free survival as the primary endpoint. It tests a different product in a population defined somewhat differently, and its primary completion is estimated for November 27, 2030. The FDA’s announcement mentioned no confirmatory trial for Anktiva in papillary-only disease, and a company that already holds the label has much less commercial reason to pay for one.
What a papillary-only patient needs to know is how much recurrence Anktiva prevents beyond what BCG and the surgeon’s resection already accomplish. Nobody can say. In January the FDA told ImmunityBio it could not tell whether the trial’s long-term outcomes reflected the drug or the disease, and the October label did not change those numbers. Four in five cohort B patients still had their bladders at three years, and a patient weighing that against surgery is entitled to the option, which NCCN had already endorsed. They are also entitled to the number nobody has measured.
R. Angelo de Claro, who directs the FDA’s Oncology Center of Excellence, said in the announcement, “We listen to patients, clinicians, and researchers, and we act when the science supports a new approach.” The agency added that it is working toward finalizing its draft guidance, which it intends to include discussion of using CIS evidence to support approval in papillary-only disease. On the day the FDA approved the first drug on that basis, the guidance was still a draft.
Sources
- FDA: FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer
- FDA: Expanded indication for nogapendekin alfa inbakicept-pmln, efficacy and dosing
- Oncology Nursing News: FDA expands Anktiva approval to papillary-only BCG-unresponsive NMIBC
- Covington: FDA advertising and promotion enforcement update, April 2026
- RAPS: FDA updates bladder cancer drug guidance
- PharmExec: FDA issues refusal-to-file letter for ImmunityBio sBLA
- OncLive: Papillary sBLA resubmitted to FDA
- ImmunityBio: FDA accepts papillary sBLA, PDUFA date January 6, 2027
- Pharmaphorum: ImmunityBio warned by FDA over misleading Anktiva content
- ClinicalTrials.gov: SunRISe-5 (NCT06211764)
- ImmunityBio: second-quarter 2026 results, SEC exhibit 99.1
- Altor BioScience: Anktiva wholesale acquisition cost disclosure