Why Japan’s review of Ferring’s bladder cancer gene therapy candidate could reshape urology

Ferring Pharmaceuticals Co., Ltd., the Japan-based subsidiary of Switzerland’s Ferring Pharmaceuticals, confirmed that Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has formally accepted its New Drug Application (NDA) for nadofaragene firadenovec, a non-replicating intravesical gene therapy for high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC). The NDA was filed on August 27, 2025, and the regulator’s acceptance signals that the therapy has cleared the initial threshold for scientific and regulatory review.

This development is more than just another regulatory milestone. It marks the first NDA filing in Japan for a non-chemotherapy, gene therapy-based approach to bladder cancer, an indication historically dominated by intravesical BCG therapy and, in cases of treatment failure, by radical cystectomy. The review process now places Japan alongside the United States, where the therapy is already approved by the U.S. Food and Drug Administration (FDA) and commercially available.

How does nadofaragene firadenovec work and why is it different from conventional therapies?

Unlike chemotherapy or immunotherapy agents administered systemically, nadofaragene firadenovec is delivered directly into the bladder via catheter once every three months. Its active mechanism involves a non-replicating adenoviral vector encoding the interferon alfa-2b gene, which transforms bladder lining cells into temporary “factories” producing high concentrations of interferon protein. This localised gene therapy creates an anti-tumour environment, amplifying the body’s natural cancer-fighting response without the systemic toxicity linked to traditional chemotherapy.

The therapy’s design allows for quarterly dosing, eliminating the burden of weekly or bi-weekly treatments, and significantly reduces the need for re-induction protocols. For patients, this means fewer hospital visits, reduced costs, and potentially higher compliance.

What did the Japanese Phase 3 trial reveal about efficacy and safety?

Ferring’s NDA submission is anchored in a Phase 3 clinical trial conducted in Japan involving 20 high-risk NMIBC patients with carcinoma in situ (CIS), with or without papillary lesions.

At the three-month mark following a single dose, 75% of patients achieved a complete response (CR). Importantly, no patient required additional re-induction, highlighting the treatment’s efficiency and simplicity compared to existing regimens. Adverse events were limited to Grade 1 (84.2%) or Grade 2 (15.8%), with no Grade 3–5 events reported, underscoring a highly favourable safety profile.

The results were presented at the 112th Annual Meeting of the Japanese Urological Association (JUA) in April 2025, where urologists noted that the therapy could redefine the country’s NMIBC treatment protocols if approved.

How do the Japanese results compare with U.S. and real-world data?

The Japanese data align closely with findings from the United States, where Mayo Clinic researchers reported a 79% complete response rate under real-world conditions. Between late 2023 and October 2024, 45 U.S. patients across three Mayo Clinic sites received nadofaragene firadenovec. Among the 29 patients analysed, 72% achieved a complete response or remained free of high-grade recurrence at the three-month mark. At six months, 62% of patients sustained this benefit, underscoring the therapy’s durability. Importantly, 94% of the group were able to avoid undergoing radical cystectomy, while 100% were alive at the six-month follow-up.

Earlier U.S. Phase 3 results, published on ClinicalTrials.gov (NCT02773849), tracked 157 patients over five years, showing an 80% overall survival rate and nearly 50% cystectomy-free survival. These outcomes provided the foundation for the FDA’s approval of nadofaragene firadenovec in 2022, making it the world’s first approved intravesical gene therapy for NMIBC.

Why is the Japanese market especially important for Ferring’s strategy?

Bladder cancer is the 13th most common cancer in Japan and the ninth most common globally, with 75% of new cases classified as NMIBC. According to Japan’s cancer registry, recurrence rates after BCG therapy exceed 50% within one year, leaving a large patient pool with limited treatment options.

Japan’s current urological guidelines primarily recommend radical cystectomy — a surgery with profound quality-of-life implications — or clinical trial participation for BCG-unresponsive patients. By offering a bladder-sparing alternative, Ferring is positioning nadofaragene firadenovec to fill a significant therapeutic gap.

For Ferring, Japan also represents a gateway to wider Asian adoption, particularly in markets where access to complex systemic immunotherapies like checkpoint inhibitors is limited due to cost or infrastructure.

What are experts and urologists saying about the therapy?

Japanese investigators, including urologists from Kochi Medical School, highlighted that nadofaragene firadenovec gives patients “a realistic chance at preserving their bladders without relying on chemotherapy.” They pointed to the convenience of quarterly dosing as a key differentiator for both physicians and patients.

At the corporate level, Ferring executives described the PMDA acceptance as a validation of their commitment to uro-oncology innovation. The company framed the therapy as a potential backbone treatment for NMIBC, stressing that patients have endured “decades of little progress” in this space.

What does this mean for the global competitive landscape in bladder cancer treatment?

The NMIBC space has historically lacked innovation compared to advanced bladder cancer, where checkpoint inhibitors like pembrolizumab (Keytruda) and nivolumab (Opdivo) have achieved broader traction. With nadofaragene firadenovec, Ferring is effectively carving out a first-mover advantage in gene therapy for urology.

Competition remains limited. While other intravesical agents and investigational gene therapies are in development, none has achieved the scale of data or regulatory approval that Ferring now holds across the U.S. and potentially Japan. Analysts suggest that if Japan grants approval in 2026, the therapy could rapidly become the standard of care for BCG-unresponsive NMIBC.

What is the investor and institutional sentiment on Ferring’s latest milestone?

Ferring Pharmaceuticals is a privately held biopharma company headquartered in Saint-Prex, Switzerland, meaning its milestones do not directly affect public markets. However, the acceptance has implications for the broader biotech investment landscape.

In the U.S., publicly traded bladder cancer players such as Sesen Bio (NASDAQ: SESN, before its 2022 merger) and ImmunityBio (NASDAQ: IBRX) have attempted to bring BCG-unresponsive therapies to market, with varying levels of success. Institutional investors tracking the uro-oncology field are closely watching how Ferring’s gene therapy shifts clinical practice, as its success could validate gene therapy approaches beyond oncology, reinforcing confidence in private and public sector investments.

Japanese investors also see potential spillover benefits. A local approval could accelerate licensing deals, contract manufacturing opportunities, and academic collaborations in Japan’s biotech ecosystem.

What comes next for Ferring and nadofaragene firadenovec in Japan?

The PMDA review typically spans 12–18 months, meaning a decision could be expected by late 2026. If approved, nadofaragene firadenovec would become Japan’s first approved bladder-sparing, non-chemotherapy gene therapy for NMIBC.

Looking ahead, analysts anticipate Ferring will expand clinical collaborations in Asia and possibly seek reimbursement pathways under Japan’s national health insurance system — a critical step for broad adoption. The therapy’s quarterly dosing model could also make it highly compatible with Japan’s resource-constrained healthcare infrastructure.

For patients and urologists, the therapy offers more than convenience. It represents the possibility of maintaining bladder function, reducing surgery burden, and improving long-term survival outcomes in a disease that has seen little therapeutic innovation in decades.

What does Ferring’s PMDA review milestone mean for the future of bladder cancer treatment in Japan and beyond?

By securing PMDA acceptance of its NDA filing for nadofaragene firadenovec, Ferring Pharmaceuticals has advanced closer to establishing the therapy as a new global standard of care for BCG-unresponsive NMIBC. With strong clinical data, robust safety outcomes, and consistent real-world validation, the therapy’s potential extends far beyond Japan, signalling a paradigm shift in urological oncology.

As the PMDA review unfolds, the biotech community, clinicians, and patients alike will be watching closely. For now, the message is clear: gene therapy is no longer a future concept in bladder cancer — it is here, tangible, and poised to change practice.