FDA extends ImmunityBio's Anktiva to papillary-only bladder cancer without a new trial

The US Food and Drug Administration on 14 October 2026 widened the label of Anktiva (nogapendekin alfa inbakicept-pmln), given with BCG, to cover adults with high-grade papillary-only non-muscle invasive bladder cancer (NMIBC) that has stopped responding to BCG ¹. The approval went to Altor BioScience, an indirect wholly owned subsidiary of ImmunityBio ².

What the FDA decided

The decision rests on extrapolation rather than a fresh efficacy readout. Anktiva was already approved for BCG-unresponsive NMIBC with carcinoma in situ (CIS), with or without papillary tumours, and the FDA has now concluded that the efficacy shown in that group can be applied to patients whose disease is high-grade papillary tumours alone ². The FDA calls Anktiva the first treatment it has approved specifically for BCG-unresponsive, high-grade papillary-only disease, and describes the decision as a new approach to evaluating therapies in this setting ¹.

The evidence carried over comes from Cohort A of QUILT-3.032, a single-arm, multicentre trial in 77 patients with BCG-unresponsive, high-risk NMIBC with CIS ². The complete response rate was 62% (95% CI: 51, 73). Among complete responders, 58% kept the response for at least 12 months and 40% for at least 24 months ².

The agency set out why it was prepared to bridge the two groups. CIS and high-grade papillary tumours grow differently, but both are aggressive urothelial cancers, often occur together, can be hard to tell apart under the microscope and, once BCG has failed, carry a high risk of recurrence and progression ². "We listen to patients, clinicians, and researchers, and we act when the science supports a new approach," said R. Angelo de Claro, director of the FDA's Oncology Center of Excellence ¹.

Dosing is unchanged: 400 mcg instilled into the bladder with BCG weekly for 6 weeks as induction, followed by maintenance courses, with a maximum treatment duration of 37 months ². The most common adverse reactions, at 15% or more, included raised creatinine, painful urination, blood in the urine, urinary frequency and urgency, urinary tract infection, raised potassium, musculoskeletal pain, chills and fever ².

What it means for the market

For ImmunityBio, the decision opens a second patient group in BCG-unresponsive NMIBC without the cost and time of a dedicated efficacy trial in papillary-only disease ¹². For other bladder cancer developers, the more consequential part is regulatory. The FDA says it is working towards finalising its draft guidance on developing drugs and biological products for BCG-unresponsive NMIBC, and that the guidance is meant to discuss using evidence from CIS studies to support approval in high-grade papillary-only disease ¹. Dr de Claro said the approach "may also help guide the development of future treatments" ¹.

The FDA frames the shared features of the two tumour types as the basis for applying CIS evidence to papillary-only disease, which gives sponsors with CIS data a stated route to a broader label in this setting ¹². The sources do not say when the guidance will be finalised, how payers will treat the expanded label, or whether European or UK regulators would accept the same reasoning.

Caveats

The supporting data come from a single-arm trial of 77 patients, and the FDA statement on the expansion cites no efficacy readout in a papillary-only population ². The prescribing information warns that delaying removal of the bladder can allow the cancer to spread, which can be fatal ¹. Treatment should be stopped if disease persists after a second induction course, recurs or progresses, or if side effects become unacceptable ². Full prescribing information for the expanded indication is due to be posted on Drugs@FDA ².