ANKTIVA® Approved in Macau for BCG-Unresponsive Bladder Cancer – First Asia Authorization

ImmunityBio’s ANKTIVA® Receives First Asian Approval in Macau, Offering New Hope for Bladder Cancer Patients

The Pharmaceutical Administration Bureau (ISAF) of Macau SAR, China, has granted regulatory approval for ANKTIVA® (nogapendekin alfa inbakicept-pmln) in combination with Bacillus Calmette-Guérin (BCG) for adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. This marks the first regulatory authorization for the immunotherapy in Asia, representing a significant step in expanding access to novel treatment options for a challenging cancer.

Addressing an Unmet Need in Bladder Cancer Treatment

NMIBC is the most common form of bladder cancer, and while initial treatment with BCG is often effective, a substantial proportion of patients experience recurrence or become unresponsive to the therapy. The development of effective alternatives to radical cystectomy – the complete removal of the bladder – is a critical area of focus, as surgery carries significant risks and impacts quality of life. Patients who are medically unfit for surgery or prefer to avoid it currently have limited options.

The approval of ANKTIVA addresses this unmet need by providing a new combination therapy designed to stimulate the immune system to fight cancer cells within the bladder. CIS, in particular, is an aggressive form of NMIBC associated with a high risk of progression, making new treatment options especially valuable.

Reliance-Based Review Accelerates Access

The Macau authorization was facilitated by a reliance-based review pathway, a growing trend in pharmaceutical regulation. This approach allows regulatory agencies to leverage the rigorous evaluations already conducted by established bodies like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). By considering these prior assessments alongside local requirements, regulators can expedite access to innovative therapies while maintaining independent oversight. This strategy is particularly beneficial for smaller regulatory authorities seeking to efficiently evaluate and approve promising new treatments.

How ANKTIVA Works: A Novel Immunotherapy Approach

ANKTIVA represents a first-in-class interleukin-15 (IL-15) receptor agonist. Unlike immune checkpoint inhibitors, which work by releasing brakes on the immune system, ANKTIVA actively stimulates the expansion and activation of immune cells. Specifically, it promotes the proliferation of natural killer cells and CD8-positive cytotoxic T lymphocytes – key players in the body’s fight against cancer. This dual action, engaging both innate and adaptive immunity, aims to enhance antitumor activity within the bladder microenvironment when used in conjunction with BCG.

Clinical Trial Data Supports Approval

The approval in Macau is based on data from the QUILT-3.032 clinical trial, which demonstrated promising results in patients with BCG-unresponsive NMIBC with CIS, with or without papillary tumors. The trial reported a complete response rate of 71 percent and a median duration of response of 26.6 months, findings published in both NEJM Evidence and The Journal of Urology. These durable responses are particularly encouraging in a disease setting characterized by high recurrence rates.

Strategic Implications for ImmunityBio and the Asia-Pacific Region

While Macau has a relatively small population, this approval is strategically important for ImmunityBio. It establishes a foothold for ANKTIVA in Asia and could pave the way for regulatory approvals in other countries within the Asia-Pacific region. Dr. Patrick Soon-Shiong, founder and Executive Chairman of ImmunityBio, described the approval as a “paradigm change” in treating the immune system, signaling the company’s ambition to expand global access to the therapy. The success of the reliance-based review process in Macau may also encourage other Asian regulators to adopt similar pathways, further accelerating the availability of innovative cancer treatments.

The long-term impact of ANKTIVA in Macau will depend on factors such as clinical adoption, reimbursement policies, and real-world outcomes. However, this approval represents a significant advancement for patients with BCG-unresponsive NMIBC, offering a potentially bladder-preserving alternative and a renewed sense of hope.