The US Food and Drug Administration (FDA) has awarded a label expansion to ImmunityBio-owned Altor Bioscience’s bladder cancer therapy, Anktiva (nogapendekin alfa inbakicept-pmln), three months ahead of its original PDUFA date, making it the first drug approved for patients with high-grade papillary-only disease.
Following the FDA’s blessing, US patients with BCG-unresponsive high-grade, papillary-only non-muscle invasive bladder cancer (NMIBC) can now receive treatment with the interleukin-15 receptor agonist. This extends the use case for Anktiva, which was first approved in 2024 alongside the Bacillus Calmette–Guérin (BCG) vaccine for the treatment of patients with BCG-unresponsive NMIBC with carcinoma in situ (CIS).
Anktiva’s expanded approval stems from the positive outcomes of the Phase III QUILT-3.032 study (NCT03022825) in which the drug triggered a 62% complete response (CR) rate in the FDA-label population, with 40% of these individuals maintaining their response at the 24-month mark.
It also marks a change in tack strategy-wise for the FDA’s review of NMIBC drugs, as the agency primarily leaned on data that had led to Anktiva’s earlier approval in a separate patient population.
According to Dr R. Angelo de Claro, director of the FDA’s Oncology Center of Excellence, this was made possible through the regulator’s review of clinical and laboratory evidence, which proved the similarities between carcinoma in situ (CIS) and the high-grade papillary-only NMIBC. Because of this overlap, the FDA was able to use data from patients with CIS pathology to inform its decision.
This approach, Claro says, could also potentially help guide the development of future treatments in NMIBC. To support this, the agency is currently working on finalising a draft guidance on developing drugs for BCG-unresponsive NMIBC, which aims to lay out a framework for sponsors who want to use evidence from patients with CIS to support potential approvals for individuals with high-grade, papillary-only disease.
Analysts tout Anktiva's 'first-mover' advantage
Anktiva’s expanded approval in NMIBC gives it a leg-up on its current competitors in the BCG-unresponsive patient population, as it is now the only drug to have secured the regulatory green light specifically for the treatment of high-grade papillary-only cases.
According to Jefferies analysts, Anktiva's new label expansion could double the current addressable NIMBC population as a first mover, while "setting a regulatory precedent for competitors." They add that the drug's on-label approval will also "broaden use beyond early adopters and may improve commercial payer coverage."
The FDA had originally requested ImmunityBio to run a separate Phase III trial for expansion into this indication, but the pair later agreed this would not be required. Johnson & Johnson, however, is already running a Phase III trial, the Sunrise-5 study, of its NMIBC therapy Inlexzo (gemcitabine intravesical system) after it was also told by the agency this was required for an expansion into high-grade papillary-only cases.
Currently, there are only a few on-patent drugs approved to treat the BCG-unresponsive subset of NMIBC patients with CIS, including Ferring Pharmaceuticals’ gene therapy, Adstiladrin (nadofaragene firadenovec‐vncg), MSD’s Keytruda (pembrolizumab) and Inlexzo.
Currently, GlobalData, parent company of Pharmaceutical Technology, estimates that Anktiva will reach blockbuster status in 2029, while going on to generate north of $4bn in global sales for ImmunityBio in 2032.
