FDA to Review Nogapendekin Alfa in Bladder Cancer and Lymphopenia
A supplemental biologics license application has been submitted to the FDA for nogapendekin alfa plus Bacillus Calmette-Guérin in papillary non-muscle invasive bladder cancer.
US FDA
- ImmunityBio has submitted a supplemental biologics license application (sBLA) to the FDA seeking approval for nogapendekin alfa inbakicept (Anktiva) plus Bacillus Calmette-Guérin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), specifically for papillary disease.
- An Expanded Access Protocol (EAP) has also been submitted to the FDA for nogapendekin alfa in the treatment of lymphopenia, a critical unmet need in cancer patients undergoing various therapies, following Regenerative Medicine Advanced Therapy (RMAT) designation for this indication.
- These submissions highlight the potential of nogapendekin alfa to significantly impact the management of both NMIBC and treatment-related complications like lymphopenia.
ImmunityBio, Inc. announced multiple regulatory submissions to the FDA, including expanding the utility of nogapendekin alfa, an innovative immunotherapy, in BCG-unresponsive NMIBC with papillary disease and for the treatment of lymphopenia in patients with cancer.1
Currently, nogapendekin alfa in combination with BCG is approved for BCG-unresponsive carcinoma in situ (CIS) with or without papillary disease.2 The approval was supported by positive results from a series of studies, including the QUILT 3.032 trial (NCT03022825).
The rationale for this application is also supported by compelling efficacy data from the QUILT 3.032 trial, demonstrating durable complete remissions in patients with BCG-unresponsive NMIBC papillary disease treated with the combination therapy.1 Notably, the submitted data indicate a high probability of bladder preservation, with 88% and 82% of patients avoiding radical cystectomy at 2 and 3 years, respectively, following treatment.
Realistic illustration of immune cells - Generated with Adobe Firefly
Further supporting these findings, the pivotal study published in the New England Journal of Medicine Evidence reported a disease-free survival (DFS) rate of 55% at 12 months, 51% at 18 months, and 48% at 24 months in patients with papillary NMIBC treated with BCG plus nogapendekin alfa.3 Moreover, this treatment regimen resulted in a 93% avoidance of cystectomy with a median follow-up of 20.7 months.
These results suggest that the combination immunotherapy, where nogapendekin alfa appears to rescue BCG efficacy in patients who have failed prior BCG treatment, could offer an effective bladder-sparing therapeutic option for papillary NMIBC.
EAP for Nogapendekin Alfa
In a separate development, ImmunityBio has submitted an EAP to the FDA for nogapendekin alfa in the treatment of lymphopenia.
Lymphopenia, characterized by a reduction in circulating lymphocytes such as natural killer cells and T cells, is a common and often severe consequence of cancer itself and various cancer treatments, including chemotherapy, radiation, steroids, and checkpoint inhibitors. This depletion of key immune cells can compromise the body's ability to fight the cancer and increase the risk of infections and other complications.
Currently, there are no approved treatments specifically targeting lymphopenia in the context of cancer therapy. The FDA previously granted RMAT designation to nogapendekin alfa for this indication in the first quarter of 2025.4
The submitted EAP, pending authorization, can provide early access to nogapendekin alfa for patients and physicians seeking a therapeutic intervention to mitigate lymphopenia that is caused by standard cancer treatments.1
The development of nogapendekin alfa serves as a potential "BioShield" against the adverse events of cancer therapies. By preserving or restoring lymphocyte counts, nogapendekin alfa could potentially improve treatment tolerance, enhance antitumor immune responses, and ultimately lead to better outcomes for patients with cancer who are undergoing various modalities of care.
