Following a bumpy regulatory road that involved a refusal-to-file, ImmunityBio has won FDA approval to expand Anktiva into papillary-only non-muscle invasive bladder cancer (NMIBC).
Wednesday’s approval comes nearly three months ahead of the FDA’s target decision date. The go-ahead covers Anktiva, given with Bacillus Calmette-Guérin (BCG), in BCG-unresponsive NMIBC patients with high-grade papillary-only tumors. The drug’s original FDA approval in 2024 is in patients with carcinoma in situ (CIS) with or without papillary tumors.
To expand Anktiva’s label, the FDA adopted a new approach to how it evaluates treatments for BCG-unresponsive NMIBC, the agency noted in an Oct. 14 press release. The new regulatory path essentially allowed ImmunityBio to bypass an expensive phase 3 trial that competitor Johnson & Johnson is currently having to grind through.
“We listen to patients, clinicians, and researchers, and we act when the science supports a new approach,” Angelo de Claro, M.D., director of the FDA’s Oncology Center of Excellence, said in a statement.
Anktiva now marks the first treatment specifically approved for BCG-unresponsive, high-grade papillary-only NMIBC. As de Claro noted, the agency drew on the similarities between NMIBC CIS and papillary-only disease to reach a positive decision on Anktiva.
In its approval announcement, the FDA again cited cohort A of ImmunityBio’s pivotal Quilt-3.032 trial in 77 patients with NMIBC CIS, with or without papillary disease. A 62% complete response rate, a 48% 12-month duration of response rate among complete responders and a 40% 24-month rate in this cohort earned the Anktiva-BCG regimen its original approval in 2024.
“While high-grade papillary and CIS tumors have different architectural growth features, both are considered aggressive urothelial carcinomas, frequently coexist, [and] can be difficult to distinguish pathologically,” the FDA noted in another Oct. 14 announcement.
“Based on the overlapping clinical and non-clinical features of these two histologic types, the FDA concluded that the prior demonstration of efficacy in patients with BCG-unresponsive NMIBC containing CIS could be extrapolated to patients with BCG-unresponsive high-grade papillary disease,” the agency said.
The FDA’s decision to review Anktiva’s profile across the two disease subtypes followed an FDA workshop in May. During the discussion, experts stated that NMIBC CIS and papillary are similar, and doctors said they treat patients with papillary-only disease using FDA-approved CIS therapies off label, according to ImmunityBio.
The two FDA announcements on Oct. 14 did not mention cohort B of the Quilt-3.032 trial in 80 patients with papillary-only NMIBC, which ImmunityBio previously said supported its supplemental application to expand Anktiva’s label. This part of the study met its primary endpoint with a 12-month disease-free survival rate of 58.2%.
However, the FDA had initially told ImmunityBio that because disease-free survival is a time-to-event endpoint, a randomized trial is required to seek approval in the papillary-only setting.
Apparently having received the same feedback from the FDA, J&J decided to run a randomized phase 3 trial dubbed Sunrise-5 to pursue a papillary indication for its rival bladder cancer therapy Inlexzo. In its third-quarter investor update Tuesday, J&J listed Sunrise-5 among phase 3 readouts expected this year.
Inlexzo got its NMIBC CIS approval in September 2025. Now, the FDA’s renewed stance suggests the phase 3 papillary disease trial, launched in April 2024, is not necessary. Inlexzo is a drug-device combo for extended release of the chemotherapy gemcitabine, whereas Anktiva is an IL-15 immunotherapy.
It wasn’t all smooth sailing for ImmunityBio, either, following some dramatic regulatory back-and-forth.
The FDA issued a refusal-to-file letter in May 2025, shooting down ImmunityBio’s expansion bid without a review. ImmunityBio appeared surprised, saying the rejection went against “unanimous guidance and encouragement” from FDA leadership at an in-person meeting earlier that year. Per ImmunityBio, FDA “unambiguously invited” the company to “expeditiously submit” the papillary indication for approval. However, Stat, citing people with knowledge of the FDA’s side of the story, later reported that the agency never endorsed the company’s plan. Instead, the agency flagged that an application would be problematic, pointing to its lack of a randomized control group, Stat reported.
The finger-pointing also happened when the FDA appeared to have raised its regulatory bar under former commissioner Marty Makary, M.D.
At the beginning of 2026, ImmunityBio Chairman Patrick Soon-Shiong also went on a podcast hosted by former White House press secretary Sean Spicer, saying he had met with President Trump’s chief of staff, Susie Wiles, health secretary Robert F. Kennedy Jr., and Makary. The company then refiled the application in March, which was accepted by the FDA in May with a PDUFA date set for Jan. 6, 2027.
In its Oct. 14 releases, the FDA said it’s finalizing draft guidance on the development of drugs for BCG-unresponsive NMIBC, which will include discussion of using evidence in CIS patients to support approval in papillary-only disease.
Meanwhile, ImmunityBio and J&J continue their fight on the commercial market. During its third-quarter update, J&J management disclosed nearly $200 million in Inlexzo sales so far this year, including “north of $90 million” in the third quarter as it took up nearly 1 in 3 eligible new patient starts. Meanwhile, ImmunityBio reported $95 million in product revenue in the first half of 2026.
As J&J’s new pharma chief Tom Cavanaugh pointed out on the call, the CIS indication is relatively small, representing roughly 4,000 patients, whereas the Sunrise-5 trial is targeting a market of roughly 15,000 patients. In addition, J&J is conducting its Sunrise-3 trial in the BCG-naïve setting, which is a population of 50,000.