Latest news with #FerringPharmaceuticals
Reuters
3 days ago
- Business
- Reuters
In-House Counsel File Practical Law The Journal
Career in Brief Experience: 2014–present: Verizon Communications Inc. (2017–present: Associate General Counsel & Director, Policy, Corporate Social Responsibility, Information Security, Legal and Security Department; 2014–2017: Assistant General Counsel & Director, Public Policy, Law and Security Department); 2011–2013: Ferring Pharmaceuticals Inc., Assistant General Counsel; 2010–2011: Mylan Specialty L.P. (formerly Dey Pharma, L.P.), Associate Counsel; 2009–2010: Biomet, Inc., Corporate Counsel, Commercial Transactions; 2002–2008: Schering-Plough Corporation, Senior Counsel; 2000–2002: Update Legal, Inc., Attorney. Education: 2000: J.D., Thurgood Marshall School of Law; 1997: Comparative Law studies, University of Oxford; 1993: H.B.A. (Industrial Relations and Economics), University of Toronto. Location of Company HQ: New York City. Primary Industry Sector: Technology and Telecommunications. Revenues in the Last Financial Year: $134.8 billion. Number of Attorneys Worldwide: 410 attorneys. Legal Department Locations: Basking Ridge, New Jersey; Alpharetta, Georgia; Irving, Texas; Washington, DC; Dublin, Ireland; Readington, England. How is the legal function structured? The legal function is structured by practice area, such as litigation, regulatory, compliance, employment, information technology, and security, as well as by business area, such as procurement, marketing, and sales. What is the top area of focus for the legal department? We are focused on delivering quality, ethical support to the business units to realize company-wide goals while remaining compliant with the Verizon Code of Conduct and industry regulations. It is imperative to remain apprised and aware of changing legal requirements. The legal department is agile, knowledgeable, and conscientious about being compliant. For example, any changes to employment laws are reviewed and implemented in compliance with industry requirements. What role do you play in Verizon's compliance program given the ever-changing regulatory environment? I make sure to stay up to date on the various regulatory requirements to effectively advise the different groups I support, including by taking training courses on the latest developments affecting our organization. How do you manage privilege and confidentiality concerns when communicating with employees? Employees in certain key areas sign non-disclosure agreements with respect to material, non-public information. Any privileged communications are marked as such when communicating with employees. Verizon maintains strict controls over the privacy of its information when disseminating the same, whether internally or externally with partners. What are some approaches that Verizon has taken to prioritize diversity? Verizon hires employees from various communities and geographies that reflect its customer base. There are several employee resource groups (ERGs) for different groups of employees, such as veterans, disabled or neurodivergent individuals, women, Latin Americans, South and East Asians, and African Americans, so that employees feel valued, appreciated, and heard. Each one of these ERGs is sponsored by a senior executive. As law departments' responsibilities evolve, what skills or competencies should in-house counsel develop to support the company's legal and operational needs? In-house counsel should develop a solid understanding of how businesses operate in specific industries, as well as an in-depth knowledge of financial principles and regulatory requirements and guidelines. What role do you see AI playing in the legal field over the next few years? Even now, I see AI becoming prevalent in the legal field, including in facilitating daily work operations such as accessing, analyzing, and utilizing information efficiently. AI cannot be avoided, but controls and parameters need to be established to prevent the unlawful dissemination of information and protect the privacy of customer information. What three things does a law firm need to do to impress you? A law firm needs to: What advice would you give to a prospective in-house counsel? Be responsive and communicative. Additionally, complete tasks thoroughly and on time in compliance with business needs and goals. What is a challenge you have faced in your role and what did you learn from it? One challenge I faced was learning to reconcile urgent business requests with established processes and protocols to remain compliant with the Verizon Code of Conduct. I learned that consistent compliance establishes known standards and this, in turn, makes legal review more efficient. If not an attorney, what would you wish to be? A motivational speaker. What are your hobbies? Traveling, fundraising for non-profits, and mentoring youth. What is your favorite book? Ruth Bader Ginsburg: A Life by Jane Sherron de Hart.
Business Wire
05-05-2025
- Health
- Business Wire
Ferring Presents New Phase 3b Safety and Effectiveness Data for Recurrent
PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals presented initial findings from the investigational Phase 3b multi-center, single-arm CDI-SCOPE study evaluating the safety and effectiveness of REBYOTA ® (fecal microbiota, live – jslm) when administered by colonoscopy. REBYOTA is the first microbiome-based therapy approved for the prevention of recurrent Clostridioides difficile (C. diff) infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for C. diff infection. These data were presented at Digestive Disease Week 2025 (DDW2025) from May 3-6 in San Diego, CA. The single-arm CDI-SCOPE study demonstrated that treatment with REBYOTA administered by colonoscopy was safe. Only 5 treatment-emergent adverse events (TEAEs) – occurring in 9.8% (4/41) of participants – were considered possibly related to REBYOTA, all of which were mild in intensity and related to the gastrointestinal tract. Two participants withdrew consent and did not complete the 8-week follow-up assessment. "Colonoscopies are routinely used by physicians managing patients with recurrent C. difficile infection," said Dr. Paul Feuerstadt, M.D., F.A.C.G., A.G.A.F., Yale School of Medicine and PACT-Gastroenterology Center, a lead investigator of CDI-SCOPE. "The insights gained from this investigative study point to administration via colonoscopy as a promising potential option for physicians to consider for their patients." A second abstract from the CDI-SCOPE study reported the burden of rCDI on health-related quality of life (HRQoL) and any improvements after treatment among 73.2% (30/41) participants. Prior to REBYOTA treatment, the most experienced rCDI symptoms were diarrhea (100%, 30/30), abdominal pain (70.0%, 21/30), and fatigue or weakness (46.7%, 14/30). All participants (100%, 30/30) reported that CDI recurrence impacted their daily living, including disrupting their sleep and forcing them to be near a bathroom at all times. Most participants also said that rCDI affected their social life and relationships (96.7%, 29/30) and their emotional wellbeing (93.3%, 28/30). Following treatment with REBYOTA, 89.7% of participants (26/29) saw an improvement in their symptoms within the first month, and more than half (51.7%, 15/29) felt an improvement within the first week. Symptom severity rating scores (0-10, none to worst) decreased significantly at Week 8 for diarrhea (from 8.9 to 1.3), abdominal pain (from 7.5 to 1.6), and fatigue (from 8.1 to 1.7). 'Recurrent CDI is a life-altering condition that many patients in our CDI-SCOPE trial called 'a literal living nightmare' that makes it impossible to function,' said Raza Ahmed, MD, Senior Director of Medical Affairs, Ferring Pharmaceuticals. 'We are proud that REBYOTA has become an important, therapy for many patients who, for too long, were stuck in an agonizing cycle of CDI recurrence. With the data we are presenting this week at DDW, Ferring is continuing its commitment to supporting patients living with rCDI and their physicians by broadening the breadth of evidence.' A third CDI-SCOPE abstract showed that 90% of the physicians who completed the 41 colonoscopy procedures in the study had a 'positive' or 'very positive' experience across all aspects of administration, including the material preparation time and ease of passage through the colonoscope. One investigator reported their experience as 'somewhat negative' citing difficulty with connecting REBYOTA to the colonoscope. All 39 patients who completed 8-week visits were assessed by the physicians as either having 'much improved' or 'very much improved' on the clinical global impression – improvement scale (CGI-I). Additional Data from PUNCH™ Clinical Program Along with the data from CDI-SCOPE, Ferring also reported two analyses with data from the PUNCH™ clinical trial program. This included: Results from an integrated safety analysis of five clinical trials (n=1192) demonstrating a favorable safety profile with REBYOTA across all trials in the development program, including in participants with inflammatory bowel disease (IBD) and immunocompromising comorbidities. TEAEs were reported in 70.9% of patients who received REBYOTA, most of which were mild or moderate in severity and related to the gastrointestinal tract. Serious TEAEs were reported in 14.3% of patients receiving REBYOTA, most of which were related to rCDI and/or preexisting conditions, with few events (<1%) being considered possibly related to treatment. There was no clustering of serious TEAEs, including in comorbid subgroups. This new exploratory analysis of the PUNCH™ CD3 trial in which improvements in HRQoL were associated with changes in microbiome and metabolome composition. In a previous exploratory analysis, patients who responded to REBYOTA vs. placebo had a healthier gut microbiome composition with an increase in the relative abundance of beneficial gut microbiota (Bacteroidia and Clostridia) and a decrease in other bacteria (Gammaproteobacteria and Bacilli). This new analysis found that the greatest improvements in HRQoL – as measured using the disease-specific C. difficile Quality of Life Survey (Cdiff32), which examines physical, mental and social domains – were associated with the mental health domain, with the largest improvements associated with higher levels of Bacteroidia and Clostridia and lower levels of other bacteria. To learn more about REBYOTA and other information, please visit or About C. diff infection C. diff infection is a serious and potentially deadly infection that impacts people across the globe. The C. diff bacterium causes debilitating symptoms, such as severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea and colitis (an inflammation of the colon). 1 C. diff infection can be the start of a vicious cycle of recurrence, causing a significant burden for patients and the healthcare system. 2,3 It has been estimated that up to 35% of C. diff infection cases recur after initial diagnosis and people who have had a recurrence are at significantly higher risk of further infections. 4,5,6,7 After the first recurrence, it has been estimated that up to 65% of patients may develop a subsequent recurrence. 6,7 Antibiotics – the current standard of care for treatment of C. diff infection – treat the disease but can also be a contributing factor to the cycle of recurrence. 1 About REBYOTA REBYOTA is a pre-packaged, single-dose 150 mL microbiota suspension for rectal administration consisting of a liquid mix of up to trillions of live microbes – including Bacteroides. REBYOTA is delivered directly to the gut microbiome and is administered by a healthcare professional in one visit. INDICATION REBYOTA (fecal microbiota, live – jslm) is indicated for the prevention of recurrence of Clostridioides difficile (C. diff) infection in individuals 18 years of age and older, following antibiotic treatment for recurrent C. diff infection. Limitation of Use REBYOTA is not indicated for the treatment of C. diff infection. IMPORTANT SAFETY INFORMATION You should not receive REBYOTA if you have a history of a severe allergic reaction (e.g., anaphylaxis) to REBYOTA or any of its components. You should report to your doctor any infection you think you may have acquired after administration. REBYOTA may contain food allergens. Most common side effects may include stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%). REBYOTA has not been studied in patients below 18 years of age. Clinical studies did not determine if adults 65 years of age and older responded differently than younger adults. You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. 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. The 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 call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X. About DDW 2025 Digestive Disease Week® (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW is an in-person and online meeting from May 3-6, 2025. The meeting showcases more than 5,600 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. More information can be found at References: Centers for Disease Control and Prevention. What is C. diff? 7 Sep. 2022. Available at: Centers for Disease Control and Prevention. 2019 Antibiotic Resistance Threats Report: Clostridioides difficile. 23 Nov. 2021. Available at: Feuerstadt P, et al. Healthcare resource utilization and direct medical costs associated with index and recurrent Clostridioides difficile infection: a real-world data analysis. J Med Econ. 2020;23(6):603-609. Riddle DJ, Dubberke ER. Clostridium difficile infection in the intensive care unit. Infect Dis Clin North Am. 2009;23(3):727-743. Nelson WW, et al. Health care resource utilization and costs of recurrent Clostridioides difficile infection in the elderly: a real-world claims analysis. J Manag Care Spec Pharm. 2021 Jul;27(7):828-838. doi: 10.18553/jmcp.2021.20395. Epub 2021 Mar 11. Kelly, CP. Can we identify patients at high risk of recurrent Clostridium difficile infection? Clin Microbiol Infect. 2012;18 (Suppl. 6): 21–27. Smits WK, et al. Clostridium difficile
Business Wire
24-04-2025
- Business
- Business Wire
U.S. FDA Approves Second Drug Product Manufacturing Facility for ADSTILADRIN ® (nadofaragene firadenovec-vncg)
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 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 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 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 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: 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: ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: 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: Accessed April 22, 2025. National Cancer Institute. Cancer Statistics. Available at: 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: Accessed April 22, 2025. More information is available at the following:
Business Wire
21-04-2025
- Business
- Business Wire
Ferring Announces Initial Data from Phase 3 Trial in Japanese Patients Demonstrating 75% Complete Response Rate at 3 Months with (nadofaragene firadenovec) in BCG-unresponsive NMIBC Patients 1
SAINT PREX, Switzerland--(BUSINESS WIRE)--Ferring Pharmaceuticals presented the first results from an ongoing Phase 3 trial in Japan assessing the efficacy and safety of nadofaragene firadenovec in 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). 1 Nadofaragene firadenovec is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population. 2 The product is given once every three months. The data – presented at the 112 th Annual Meeting of the Japanese Urological Association (JUA 2025) held April 17-19 in Fukuoka, Japan – showed a complete response (CR) rate at three months of 75% (15/20) among Japanese patients with CIS ± high-grade Ta/T1. 1 Importantly, these results were achieved following a single quarterly administration, representing a significant clinical advantage. The efficacy appears higher than previously reported in the US Phase 3 trial 3,4 and adds to a growing body of evidence. Recent real-world data was presented by the Mayo Clinic and showed 79% CR rate, 2 demonstrating consistent efficacy and safety when patients are treated with nadofaragene firadenovec. Patients who responded at the three-month assessment received continued every three months doses until disease recurrence. These data will be presented at a future congress. Within this conservatively designed protocol, a replication of CS-003 US Phase 3 trial, patients who did not respond at three months were not offered a re-induction dose. This contrasts with a growing trend in other NMIBC trials, where re-induction is included in the study protocols for patients who do not achieve a response at initial assessment. Re-induction is now being explored with nadofaragene firadenovec in other trials. ' When BCG therapy is ineffective, patients are forced to choose invasive surgery, i.e., total bladder removal, but nadofaragene firadenovec may provide a new treatment option, ' noted Professor Keiji Inoue, M.D., Ph.D., Department of Urology, Kochi Medical School. ' These findings are particularly significant for Japanese patients, as our treatment options have been more limited compared to other regions. The ability to achieve such promising results represents an important advancement for our clinical practice. ' Overall, 80% of participants (16 patients) experienced drug-related adverse events (AEs), with 76 total AEs recorded. All reported AEs were either Grade 1 (64 AEs in 15 participants, 84.2%) or Grade 2 (12 AEs in 5 participants, 15.8%). No Grade 3, 4, or 5 AEs were reported, demonstrating a clinically manageable and tolerable safety profile. The data from Japan add to the growing body of evidence, including recent real-world data presented by the Mayo Clinic, 2 demonstrating favorable efficacy and safety when patients are treated with nadofaragene firadenovec. ' At Ferring, we are committed to meeting the unmet needs in bladder cancer care and equipping uro-oncologists with critical evidence they need to deliver effective, and life-changing treatment, ' said Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals. ' These new Phase 3 findings affirm the safety profile of nadofaragene firadenovec, demonstrating a three-month efficacy that is higher than previously reported in our Phase 3 clinical trial, and complements results from an ongoing independent real-world study presented earlier this year. Collectively, the data are broadening our understanding of the value that nadofaragene firadenovec offers, furthering our journey to establish nadofaragene firadenovec as the new standard of care and backbone therapy across the urothelial cancer disease spectrum.' Bladder cancer is the 9 th most common cancer worldwide by incidence, with approximately 614,000 new cases diagnosed each year. 5 Non-muscle invasive bladder cancer (NMIBC) accounts for about 75% of all newly diagnosed bladder cancers. 6 For patients who do not respond to BCG treatment, current options are limited, often leading to invasive surgery involving complete bladder removal. The strong efficacy demonstrated in this Japanese patient population suggests nadofaragene firadenovec could address a critical unmet need in the bladder cancer treatment landscape, delaying radical cystectomy and preserving quality of life for thousands of patients annually. The ongoing Phase 3 Japanese trial is evaluating the efficacy and safety of nadofaragene firadenovec in two cohorts of high-risk BCG-unresponsive NMIBC patients: those with CIS ± HG Ta/T1, and patients with papillary tumors (Ta/T1) only. Results from the cohort with CIS ± HG Ta/T1 were presented on April 19 at JUA 2025. 1 About nadofaragene firadenovec Nadofaragene firadenovec (marketed as ADSTILADRIN ® in the US) 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 cancer. Nadofaragene firadenovec has been studied in a clinical trial programme 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 (full inclusion criteria published on NCT02773849). 3 Final 60-month follow-up data demonstrated an 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 tumors (±Ta/T1), and in patients with high-grade Ta/T1 without CIS 5. Most treatment emergent AEs (AEs) at five years were transient Grade 1 or 2 (66% of all patients studied), and <4% of patients experienced Grade 3 AEs with the most common being discharge around the catheter during instillation, fatigue, bladder spasm, urgency to urinate, chills, dysuria, pyrexia, and urinary incontinence. There were no Grade 4 or 5 AEs, no treatment-related deaths, and no new safety signals reported with long-term follow-up. Ferring is leading the future in uro-oncology treatment with nadofaragene firadenovec at the centre, while expanding access with the support of new, state-of-the-art manufacturing facilities. About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body. Bladder cancer is the ninth most commonly diagnosed cancer globally. 75% of bladder cancers present as NMIBC. 7 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard of care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease. 7 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). About Ferring Pharmaceuticals Ferring Pharmaceuticals 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. Ferring was founded in 1950 and employs more than 7,000 people worldwide. 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 or connect with us on LinkedIn, Instagram, YouTube, Facebook and X. 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 th Annual Meeting of the Japanese Urological Association, April 19, 2025. Available at: 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 ADSTILADRIN | FDA 4 Boorjian SA, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2021;22(1): 107-117. 5 Narayan VM, at 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 6 World Cancer Research Fund International. Bladder cancer statistics. Available at: Last accessed: March 2025. 7 Deng S, et al. Global research trends in non-muscle invasive bladder cancer: Bibliometric and visualized analysis. Front Oncol. 2022; 12:1044830. 8 Babjuk M, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer (Ta, T1, and Carcinoma in Situ). Eur Urol. 2022;81(1):75-94.
Business Wire
21-04-2025
- Business
- Business Wire
Ferring Unveils New Data with ADSTILADRIN ® (nadofaragene firadenovec-vncg) at 112 th Annual Meeting of the Japanese Urological Association
PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals presented the initial results from an ongoing Phase 3 trial (NCT05704244) in Japan assessing the efficacy and safety of ADSTILADRIN ® (nadofaragene firadenovec-vncg) in 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). 1 ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population. ADSTILADRIN is not approved for use in Japan. The data – presented at the 112th Annual Meeting of the Japanese Urological Association (JUA 2025) held April 17-19 in Fukuoka, Japan – showed a complete response (CR) rate at three months of 75% (15/20) among participants in Japan with CIS ± high-grade Ta/T1. 1 Overall, 80% of participants (16 patients) experienced a drug–related adverse event (AE), with 76 total AEs recorded. 1 All reported AEs were either Grade 1 (64 AEs in 15 participants, 84.2%) or Grade 2 (12 AEs in 5 participants, 15.8%). 1 No grade 3, 4, or 5 AEs were reported, demonstrating a clinically manageable and tolerable safety profile. 1 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). 2 "At Ferring, we are committed to meeting the unmet needs in bladder cancer care and equipping uro-oncologists with critical evidence they need to deliver effective and life-changing treatment," said Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals. "These initial Phase 3 findings from Japan affirm the safety profile of ADSTILADRIN, demonstrating a three-month efficacy that appears to be higher than previously reported in our Phase 3 clinical trial, and consistent with results from an ongoing independent real-world study presented earlier this year. Collectively, the data are broadening our understanding of the value that ADSTILADRIN offers, furthering our journey to establish ADSTILADRIN as the new standard of care and backbone therapy across the urothelial cancer disease spectrum.' The ongoing Phase 3 Japanese trial is evaluating the efficacy and safety of ADSTILADRIN in two cohorts of high-risk BCG-unresponsive NMIBC patients: those with CIS ± HG Ta/T1, and patients with papillary tumors (Ta/T1) only. 1 Results from the cohort with CIS ± HG Ta/T1 were presented on April 19 at JUA 2025 in the oral presentation, 'Efficacy and Safety of Nadofaragene Firadenovec for BCG-Unresponsive Non–Muscle-Invasive Bladder Cancer: Initial Results From an Ongoing Japanese Phase 3 Trial.' 1 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. 3 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 NCT02773849). 3 Patients in the pivotal Phase 3 study who had no evidence of high-grade recurrence at 12 months following initial ADSTILADRIN therapy continued receiving treatment once every three months at the discretion of their treating physician, entering an additional four-year treatment and monitoring phase. Final 60-month follow-up data demonstrated an 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 tumors (±Ta/T1), and in patients with high-grade Ta/T1 without CIS. 4 Most treatment emergent AEs (TEAEs) at five years were transient Grade 1 or 2 (66% of all patients studied), and <4% of patients experienced Grade 3 TEAEs with the most common being discharge around the catheter during instillation, fatigue, bladder spasm, urgency to urinate, chills, dysuria, pyrexia, and urinary incontinence. There were no Grade 4 or 5 TEAEs, no treatment-related deaths, and no new safety signals reported with long-term follow-up. 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 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, 7 fourth among men, 8 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025. 8 Historically, 75% of bladder cancer presents as NMIBC. 9 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. 10 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). 11 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 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 call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X. References: 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 19, 2025. Available at: 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. ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: Accessed April 1, 2025. Ferring Pharmaceuticals. Final Five-Year Analysis of Phase 3 Data With ADSTILADRIN® (nadofaragene firadenovec-vncg) Shows Durable Bladder Preservation and Consistent Long-Term Safety in BCG-unresponsive NMIBC. Published May 6, 2024. Accessed April 1, 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: Accessed April 1, 2025. National Cancer Institute. Cancer Statistics. Available at: Accessed April 1, 2025. American Cancer Society. Cancer Facts & Figures 2025. Available at: Accessed April 1, 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 4.2024). Available at: Accessed April 1, 2025. More information is available at the following:
