U.S. FDA Approves Second Drug Product Manufacturing Facility for ADSTILADRIN ® (nadofaragene firadenovec-vncg)

Business Wire

24-04-2025

PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals announced today the U.S. Food and Drug Administration (FDA) approved a state-of-the-art drug product manufacturing hub in Parsippany, NJ, for its intravesical non-replicating gene therapy ADSTILADRIN ® (nadofaragene firadenovec-vncg). The approval expands Ferring's manufacturing capabilities to three cutting-edge sites dedicated to bringing ADSTILADRIN to patients and secures a final $200 million payment from Royalty Pharma (Nasdaq: RPRX) as part of a royalty-based financing agreement announced in 2023.
ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the FDA for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). Bladder cancer is the sixth most diagnosed cancer in the United States, and the majority of patients are diagnosed with non-muscle invasive disease. 1
'The FDA approval of our new manufacturing facility for ADSTILADRIN represents our unwavering dedication to delivering high-quality, innovative therapies at scale,' said Armin Metzger, Executive Vice President, Chief Technical Operations Officer, Ferring Pharmaceuticals. 'This expansion and diversification of our manufacturing footprint will further ensure stable and sustainable supply of ADSTILADRIN to meet the anticipated growth in global demand.'
Located on Ferring's U.S. campus in Parsippany, NJ, the new state-of-the-art manufacturing site features a cutting-edge manufacturing suite, fully integrated with specialized modern technology and equipment to produce another source of supply for ADSTILADRIN. The 12,000 square foot facility features renewable energy solutions such as waste heat recovery with heat pumps and solar energy, complementing Ferring's commitment to protect the environment by reducing its impact on the planet.
' ADSTILADRIN has transformed the treatment landscape for BCG-unresponsive bladder cancer patients and drives Ferring's continued growth in uro-oncology, ' said Bipin Dalmia, Global Head of Uro-Oncology and Urology Franchise, Ferring Pharmaceuticals. 'The growing body of clinical evidence for ADSTILADRIN — including the recently announced independent real-world data 2 and data from our Japan Phase 3 trial 3 — underscores the impact of this therapy. The FDA's approval of this additional manufacturing site is a testament to our commitment to make ADSTILADRIN globally available to every bladder cancer patient who needs it.'
About ADSTILADRIN
ADSTILADRIN ® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of 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 tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder's cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body's own natural defenses against the cancer.
ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment. 4 The pivotal Phase 3 study met its primary endpoint, with more than half (51%) of the 98 patients (95% CI [41%, 61%]) with CIS with or without papillary tumors (±Ta/T1) achieving a complete response (CR) by three months (full inclusion criteria published on clinicaltrials.gov: NCT02773849). 4
Earlier this month, Ferring announced results from an ongoing Phase 3 trial of ADSTILADRIN in BCG-unresponsive CIS ± high-grade Ta/T1 patients (n=20) in Japan. At 3 months, 75% (15/20) of patients achieved a complete response. 2 Overall, 80% of participants experienced a drug–related AE, all of which were either Grade 1 (84.2%) or Grade 2 (15.8%). The data from Japan add to the growing body of evidence, including recent real-world data presented by the Mayo Clinic that showed a 79% CR rate, demonstrating consistent effectiveness and safety when patients are treated with ADSTILADRIN at three months (19/24 patients with CIS +/- Ta/T1). 3
Ferring is leading the future in uro-oncology treatment with ADSTILADRIN at the center, while expanding access with the support of new, state-of-the-art manufacturing facilities. As announced in January 2024, ADSTILADRIN is fully available and accessible in the U.S. ADSTILADRIN has confirmed 99 percent coverage for commercial and government-insured patients. As of April 1, 2024, in accordance with the Centers for Medicare and Medicaid Services (CMS), ADSTILADRIN established an Average Sales Price (ASP). Since the establishment of ASP, all covered claims submitted for reimbursement have received payment within an average of 25 days. 5
About Ferring Uro-Oncology
Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at FerringUroOncology.com and on the dedicated Ferring Uro-Oncology channels on LinkedIn and X.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall. 6 In the United States, bladder cancer is the sixth most common cancer, 1 fourth among men, 7 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025. 1 Historically, 75% of bladder cancer presents as NMIBC. 8 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease. 9 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder). 10
INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of 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 tumors.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.
WARNINGS AND PRECAUTIONS:
Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.
USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.
ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).
You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.
Please click to see the full Prescribing Information.
About Ferring Pharmaceuticals
Ferring Pharmaceuticals is a privately-owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees.
For more information, please visit www.ferringusa.com, call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X.
References:
American Cancer Society. Cancer Facts & Figures 2025. Available at: https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2025/2025-cancer-facts-and-figures-acs.pdf. Accessed April 22, 2025.
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.
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 th Annual Meeting of the Japanese Urological Association, April 22, 2025. Available at: https://www.micenavi.jp/jua2025/search/detail_program/id:2055
ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: https://clinicaltrials.gov/ct2/show/NCT02773849. Accessed April 22, 2025.
Ferring Access Support benefits investigations for commercial and government-insured patients through March 15, 2024.
Urology Care Foundation. Non-muscle Invasive Bladder Cancer. Available at: https://www.urologyhealth.org/urology-a-z/n/non-muscle-invasive-bladder-cancer. Accessed April 22, 2025.
National Cancer Institute. Cancer Statistics. Available at: https://www.cancer.gov/about-cancer/understanding/statistics#:~:text=The%20most%20common%20cancers%20%28listed%20in%20descending%20order,pancreatic%20cancer%2C%20leukemia%2C%20thyroid%20cancer%2C%20and%20liver%20cancer. Accessed April 22, 2025.
Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer. Eur Urol. 2022 Jan;81(1):75-94.
Lidagoster S, et al. BCG and Alternative Therapies to BCG Therapy for Non-Muscle-Invasive Bladder Cancer. Curr Oncol. 2024 Feb 16;31(2):1063-1078. doi: 10.3390/curroncol31020079.
National Comprehensive Cancer Network. Bladder Cancer (Version 1.2025). Available at: https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf. Accessed April 22, 2025.
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