Ferring Japan announces PMDA Acceptance of NDA Filing for nadofaragene firadenovec

Nadofaragene firadenovec is the first and only non-replicating gene therapy administered intravesically, and it is already approved and launched in the USThis represents the first filing of a novel non-chemotherapy approach in non-muscle invasive bladder cancer (NMIBC) treatmentIn the Japanese Phase 3 clinical trial, 75% complete response rate was achieved with single quarterly dosing, without requiring re-induction protocols1This acceptance highlights Ferring's ongoing commitment to establish the new standard of care for BCG-unresponsive NMIBC

Ferring Pharmaceuticals Co., Ltd. announced that the PMDA has accepted the NDA for nadofaragene firadenovec for review, following submission on August 27^th, 2025. This non-replicating gene therapy, administered intravesically, offers patients with NMIBC a bladder-sparing treatment option. Nadofaragene firadenovec's quarterly dosing eliminates the burden of frequent treatments, while delivering a non-chemotherapy mechanism of action through interferon gene therapy. The NDA acceptance further highlights Ferring's ongoing commitment to establish the new standard of care for high-risk BCG-unresponsive NMIBC.

Professor Keiji Inoue, M.D., Ph.D., Department of Urology, Kochi Medical School, stated: "Nadofaragene firadenovec represents an option for those who failed NMIBC treatment. As the first choice after BCG failure, this bladder sparing gene therapy offers patients a non-chemotherapy option that transforms their own bladder cells into interferon-producing factories. The 75% complete response rate achieved with convenient quarterly dosing provides hope for patients who previously faced limited treatment options."

The NDA for nadofaragene firadenovec is based on results from a Phase 3 trial conducted in Japan.¹ The primary outcome of a complete response (CR) rate of 75% at 3 months, following a single dose in 20 high-risk Japanese patients with carcinoma in situ (CIS), with or without concomitant high-grade Ta or T1 papillary lesions, was presented at the 112^th Annual Meeting of the Japanese Urological Association (JUA, April 17-19, 2025, Fukuoka). All treatment-related adverse events were limited to Grade 1 (84.2%) or Grade 2 (15.8%), with zero Grade 3, 4, or 5 adverse events reported - confirming the therapy's favourable safety and tolerability profile.¹

These results are consistent with independent US real-world data presented by the Mayo Clinic, which demonstrated 79% complete response rate.² The results from the Mayo Clinic were achieved following a single quarterly dosing, representing clinical and convenience advantages over existing therapies, without requiring re-induction protocols.² Patients who achieved complete response continued quarterly maintenance dosing.

Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals stated "Traditionally, when BCG therapy proved insufficient, patients had no choice but to undergo highly invasive treatments such as radical cystectomy or risk cancer progression. Nadofaragene firadenovec is expected to offer a new bladder-sparing treatment option. At Ferring, we are committed to addressing the unmet needs in bladder cancer treatment by providing urologists with critical insights that enable effective, personalised, and groundbreaking therapies."

"Our ambition is to establish nadofaragene firadenovec as the new standard of care and the backbone therapy for NMIBC treatment," said Bipin Dalmia, Global Head, Uro-Oncology & Urology Franchise. "High-risk NMIBC patients who no longer respond to BCG have endured decades of little progress and currently face bladder removal as their primary option. This PMDA acceptance validates our strategic commitment to bring this treatment to Japanese patients."

About nadofaragene firadenovec

Nadofaragene firadenovec represents the first and only FDA-approved intravesical non-replicating gene therapy for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.

As the first choice therapy following BCG failure, this non-chemotherapy approach utilises a non-replicating adenovirus vector-based therapy containing the interferon alfa-2b gene. Administered locally as convenient monotherapy by catheter directly into the bladder once every three months only, the mechanism transforms bladder wall cells into interferon microfactories, creating high and transient local expression of interferon alfa-2b protein - amplifying the body's natural cancer-fighting capabilities through gene therapy rather than traditional chemotherapy approaches.

This therapeutic approach has been investigated through comprehensive clinical evaluation including 157 patients in the US with high-risk BCG-unresponsive NMIBC who demonstrated inadequate BCG response (full inclusion criteria published on clinicaltrials.gov: NCT02773849). 5-year follow-up data confirmed 80% overall survival rate and 49% cystectomy-free survival rate in adult patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumours (±Ta/T1), and in patients with high-grade Ta/T1 without CIS. Long-term safety profile maintained with most treatment emergent AEs remaining transient Grade 1 or 2 (66% of all patients studied), and 4% experiencing Grade 3 AEs.³

In the real world setting, between November 2023 and October 2024, 45 patients across three Mayo Clinic locations in the US were treated with nadofaragene firadenovec for BCG-unresponsive NMIBC. Out of 45 treated patients, 29 were included in the analysis.² After a median follow-up of 8.2 months: 72% of patients showed a complete response or were free from high-grade recurrence at 3 months. 62% maintained this response at 6 months. 94% avoided bladder removal surgery (cystectomy). 100% were still alive at 6 months.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC affects the superficial bladder layer without deeper invasion or metastatic spread. Bladder cancer represents a major clinical challenge as the 13^th most commonly diagnosed cancer in Japan and ninth globally,⁴ with 75% presenting as NMIBC in 2022.⁴ While intravesical BCG remains first-line standard care for high-risk NMIBC, over 50% of patients experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease requiring radical intervention.⁵

Current treatment options remain limited for BCG-unresponsive patients, with JUA guidelines recommending only radical cystectomy or clinical trial participation.⁶ Nadofaragene firadenovec offers the first choice non-chemotherapy gene therapy option for these patients, providing an alternative to immediate radical surgery.

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a privately owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. We are leaders in reproductive medicine with a strong heritage in gastroenterology and urology, and are at the forefront of innovation in uro-oncology gene therapy. The company is headquartered in Saint-Prex, Switzerland, and has operating subsidiaries in more than 50 countries which market its medicines in over 100 countries.

Learn more at www.ferring.com, or connect with us on LinkedIn, Instagram, YouTube, Facebook and X.

About Ferring Pharmaceuticals Co., Ltd.

Ferring Pharmaceuticals Co., Ltd. established Japanese operations in February 2001 as the dedicated Japanese subsidiary of Ferring Pharmaceuticals, headquartered in Toranomon, Minato-Ku, Tokyo. For more information, please visit Home - Ferring Japan

*As of September 2025, within Japan.

References

¹ Inoue K, Kikuchi E, Nishiyama H, Nasu Y, et al. Efficacy and Safety of Nadofaragene Firadenovec for BCG-Unresponsive Non–Muscle-Invasive Bladder Cancer: Initial Results From an Ongoing Japanese Phase 3 Trial. Presented at the 112 Annual Meeting of the Japanese Urological Association, April 19, 2025. Available at: https://www.micenavi.jp/jua2025/search/detail_program/id:2055
² Moyer J, Durant A, Nguyen M. Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. Presented at the Annual Meeting of the American Society of Clinical Oncology GU, February 2025. 
³ Narayan VM, et al. Efficacy of Intravesical Nadofaragene Firadenovec for Patients with BCG-Unresponsive Non-muscle Invasive Bladder Cancer: 5 Year Follow-Up from a Phase 3 Trial. J Urol. 2024 May 5. doi: 10.1097/JU.0000000000004020. 
⁴ World Cancer Research Fund International. Bladder cancer statistics. Available at: https://www.wcrf.org/cancer-trends/bladder-cancer-statistics/. Last accessed: March 2025. 
⁵ Deng S, et al. Global research trends in non-muscle invasive bladder cancer: Bibliometric and visualized analysis. Front Oncol. 2022; 12:1044830. 
⁶ Matsumoto H, Shiraishi K, Inoue K, et al. Clinical Practice Guidelines for Bladder Cancer 2019 update by the Japanese Urological Association: Summary of the revision. Available at: Clinical Practice Guidelines for Bladder Cancer 2019 update by the Japanese Urological Association: Summary of the revision. Last accessed: September 2025.

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