The safety and effectiveness of ADSTILADRIN (nadofaragene firadenovec-vncg) were evaluated in CS-003, an open-label, multicenter, single-arm study of 103 patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non–muscle-invasive bladder cancer, 98 of whom had BCG-unresponsive carcinoma in situ (CIS) with or without papillary tumors and could be evaluated for response. The primary endpoint was complete response (CR) in CIS ± high-grade Ta/T1 at any time after the first instillation.1
Efficacy
ADSTILADRIN Demonstrated Durability
Out to 5 Years
Study design
In the study, patients were not reinduced if a CR did not occur after the first dose
ADSTILADRIN has proven and durable
complete responses out to 5 years
3 months (primary endpoint)
51%
of the CIS cohort achieved CR by month 3 (after 1 instillation)1,2
Of the patients who achieved CR by month 3*:
AT 1 YEAR
46%
remained free of
high-grade recurrence2
high-grade recurrence2
AT 2 YEARS
36%
remained free of
high-grade recurrence3
high-grade recurrence3
AT 3 YEARS
25%
remained free of
high-grade recurrence4
high-grade recurrence4
AT 5 YEARS
11%
remained free of
high-grade recurrence5
high-grade recurrence5
Among all patients in the CIS cohort at 5 years,
96%
did not progress to muscle-invasive bladder cancer as assessed by transurethral resection of bladder tumor at recurrence (n=103/107)5,6†
AT 57 MONTHS5
25% of patients (14/55) had an ongoing response at time of last assessment
Any patient lost to follow-up or who switched therapy was considered a treatment failure
ADSTILADRIN provided bladder preservation
out to 5 years
A key patient-centered goal is bladder preservation5
Cystectomy-free survival rates for the CIS cohort (n=103):
AT 1 YEAR2
≈3/4
of patients‡
AT 5 YEARS5
≈1/2
of patients‡
*Based on patients (n=50) who achieved a CR; reflects period from the time CR was achieved.1
†As a single-arm study, low disease progression rate cannot conclusively be attributed to the therapy.5
‡The Kaplan-Meier estimated cystectomy-free survival rate for the CIS cohort was 73% at 1 year and 43% at 5 years.5
Hear From a Board-Certified Urologist
“In bladder oncology, I think that this represents a transformative breakthrough.”
Lawrence Karsh, MD, FACS, treated patients in the ADSTILADRIN clinical trial
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Form ToggleReferences: 1. ADSTILADRIN. Package insert. Ferring Pharmaceuticals, Inc; August 2024. 2. Boorjian SA, Alemozaffar M, Konety BR, 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. doi:10.1016/S1470-2045(20)30540-4 3. Schuckman AK, Lotan Y, Boorjian SA, Cilwa KE, Dinney CPN. Efficacy of intravesical nadofaragene firadenovec for patients with carcinoma in situ (CIS), BCG-unresponsive non-muscle invasive bladder cancer (NMIBC): longer-term follow-up from the phase III trial. J Urol. 2021;206(suppl 3):e296. doi:10.1097/JU.0000000000002001.01 4. Boorjian SA, Narayan VM, Konety BR, et al. Efficacy of intravesical nadofaragene firadenovec for patients with BCG-unresponsive carcinoma in situ of the bladder: 36-month follow-up from a phase 3 trial. Presented at: 24th Annual Meeting of the Society of Urologic Oncology; November 28-December 1, 2023; Washington, DC. 5. Narayan VM, Boorjian SA, Alemozaffar M, et al. Efficacy of intravesical nadofaragene firadenovec for patients with Bacillus Calmette-Guérin-unresponsive nonmuscle-invasive bladder cancer: 5-year follow-up from a phase 3 trial. J Urol. 2024;212(1):1-12. doi:10.1097/JU.0000000000004020 6. Data on file. Ferring Pharmaceuticals, Inc. July 2024.
Important Safety Information
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 1-800-332-10881-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING1-888-FERRING.
Please see full Prescribing Information for ADSTILADRIN.