AstraZeneca’s Imfinzi Granted Priority Review by FDA for Groundbreaking Perioperative Immunotherapy in Bladder Cancer

(IN BRIEF) AstraZeneca’s Imfinzi (durvalumab) has been granted Priority Review by the FDA for the treatment of muscle-invasive bladder cancer (MIBC), potentially becoming the first perioperative immunotherapy in this curative-intent setting. Supported by results from the NIAGARA Phase III trial, Imfinzi demonstrated a 32% reduction in disease progression risk and a 25% reduction in the risk of death compared to standard treatment. Safety profiles were consistent, and the therapy did not hinder surgery or chemotherapy completion. The FDA decision is expected by mid-2025, with regulatory reviews also underway in the EU, Japan, and other regions.

(PRESS RELEASE) CAMBRIDGE, 7-Dec-2024 — /EuropaWire/ — AstraZeneca’s supplemental Biologics License Application (sBLA) for Imfinzi (durvalumab) has been granted Priority Review by the US Food and Drug Administration (FDA) for the treatment of patients with muscle-invasive bladder cancer (MIBC). If approved, Imfinzi would become the first and only perioperative immunotherapy regimen available in this curative-intent setting, offering a groundbreaking advancement in the standard of care.

A Promising Step Forward for Bladder Cancer Treatment

MIBC affects approximately one in four bladder cancer patients and is associated with high recurrence rates and poor survival outcomes, even after radical cystectomy. Current standard treatments include neoadjuvant chemotherapy followed by cystectomy, but there remains a significant unmet need for therapies to improve long-term outcomes.

The FDA’s Priority Review designation recognizes Imfinzi’s potential to fill this gap by significantly delaying disease recurrence and extending survival in patients undergoing curative-intent treatment. The FDA’s decision is expected by the second quarter of 2025.

NIAGARA Trial Results

The sBLA is supported by data from the NIAGARA Phase III trial, presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in The New England Journal of Medicine. The study demonstrated that patients treated with Imfinzi in combination with neoadjuvant chemotherapy before surgery, followed by Imfinzi monotherapy, achieved superior outcomes compared to the standard regimen of chemotherapy and surgery alone.

Key findings include:

A 32% reduction in the risk of disease progression, recurrence, or death with Imfinzi (hazard ratio [HR] 0.68; p<0.0001).
At two years, 67.8% of patients receiving the Imfinzi regimen were event-free, compared to 59.8% in the comparator group.
A 25% reduction in the risk of death with Imfinzi (HR 0.75; p=0.0106). At two years, 82.2% of patients treated with Imfinzi were alive, compared to 75.2% in the comparator arm.

Safety outcomes were consistent with known profiles of the individual agents, and Imfinzi did not impact patients’ ability to complete chemotherapy or undergo surgery.

Transforming the Standard of Care

Susan Galbraith, Executive Vice President of Oncology R&D at AstraZeneca, emphasized the significance of these findings:
“Nearly half of MIBC patients experience disease progression or recurrence despite curative-intent treatment. Imfinzi’s potential to delay recurrence and improve survival represents a vital step forward in addressing this unmet need.”

Global Regulatory Progress

In addition to the FDA’s Priority Review, regulatory applications for Imfinzi are under review in the EU, Japan, and other countries based on the NIAGARA trial data. If approved, Imfinzi could redefine the treatment paradigm for MIBC, offering patients a more effective perioperative option to combat this challenging cancer.

Notes

Muscle-invasive bladder cancer
Bladder cancer is the 9th most common cancer in the world, with more than 614,000 patients diagnosed each year.6 The most common type of bladder cancer is urothelial carcinoma, which begins in the urothelial cells of the urinary tract.7 Approximately 50% of patients who undergo bladder removal surgery experience disease recurrence.5 Treatment options that prevent disease recurrence after surgery are critically needed in this curative-intent setting.

NIAGARA
NIAGARA is a randomised, open-label, multi-centre, global Phase III trial evaluating perioperative Imfinzi as treatment for patients with MIBC before and after radical cystectomy. In the trial, 1,063 patients were randomised to receive Imfinzi plus neoadjuvant chemotherapy prior to cystectomy followed by Imfinzi, or neoadjuvant chemotherapy alone prior to cystectomy with no further treatment after surgery. NIAGARA is the largest global Phase III trial in this setting.

The trial is being conducted at 192 centres across 22 countries including in North America, South America, Europe, Australia and Asia. Its dual primary endpoints are EFS and pathologic complete response. Key secondary endpoints are OS and safety.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is the only approved immunotherapy and the global standard of care in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy. Additionally, Imfinzi is approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based chemoradiotherapy; as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC; in combination with chemotherapy (etoposide and either carboplatin or cisplatin) for the treatment of extensive-stage SCLC; and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC.

In addition to its indications in lung cancers, Imfinzi is approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU.

Imfinzi is also approved in combination with chemotherapy (carboplatin and paclitaxel) followed by Imfinzi monotherapy in primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR) in the US. In the EU, Imfinzi plus chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient (pMMR) advanced or recurrent endometrial cancer, and Imfinzi plus chemotherapy followed by Imfinzi alone is approved for patients with dMMR disease. In Japan, Imfinzi plus chemotherapy followed by Imfinzi monotherapy has also been approved as 1st-line treatment in primary advanced or recurrent endometrial cancer, and Imfinzi plus chemotherapy followed by Imfinzi and Lynparza has been approved for patients with pMMR disease.

Since the first approval in May 2017, more than 374,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, breast cancer, several gastrointestinal and gynaecologic cancers, and other solid tumours.

AstraZeneca in immuno-oncology (IO)
AstraZeneca is a pioneer in introducing the concept of immunotherapy into dedicated clinical areas of high unmet medical need. The Company has a comprehensive and diverse IO portfolio and pipeline anchored in immunotherapies designed to overcome evasion of the anti-tumour immune response and stimulate the body’s immune system to attack tumours.

AstraZeneca strives to redefine cancer care and help transform outcomes for patients with Imfinzi as a monotherapy and in combination with Imjudo as well as other novel immunotherapies and modalities. The Company is also investigating next-generation immunotherapies like bispecific antibodies and therapeutics that harness different aspects of immunity to target cancer, including cell therapy and T-cell engagers.

AstraZeneca is pursuing an innovative clinical strategy to bring IO-based therapies that deliver long-term survival to new settings across a wide range of cancer types. The Company is focused on exploring novel combination approaches to help prevent treatment resistance and drive longer immune responses. With an extensive clinical programme, the Company also champions the use of IO treatment in earlier disease stages, where there is the greatest potential for cure.

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca.

References

FDA. Priority Review. Available at: https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review. Accessed December 2024.
Burger M, et al. Epidemiology and Risk Factors of Urothelial Bladder Cancer. Eur Urol. 2013;63(2):234-241.
National Collaborating Centre for Cancer. Bladder Cancer: Diagnosis and Management. London: National Institute for Health and Care Excellence (NICE). Available at: https://www.ncbi.nlm.nih.gov/books/NBK356289. Accessed December 2024.
Cerner CancerMPact database. Accessed December 2024. Reflects epidemiology estimates across G8 countries (US, EU, Japan, China).
Witjes JA, et al. EAU Guidelines on Muscle-invasive and Metastatic Bladder Cancer. Eur Urol. 2021;1-94.
World Health Organization. International Agency for Research on Cancer. Bladder Fact Sheet. Available at: https://gco.iarc.who.int/media/globocan/factsheets/cancers/30-bladder-fact-sheet.pdf. Accessed December 2024.
American Cancer Society. What Is Bladder Cancer? Available at: https://www.cancer.org/cancer/bladder-cancer/about/what-is-bladder-cancer.html. Accessed December 2024.

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SOURCE: AstraZeneca

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