FDA D.I.S.C.O. Burst Edition: FDA approval of Adstiladrin (nadofaragene firadenovec-vncg) for patients with high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors
Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer therapeutic approval.
On December 16, 2022, the FDA approved nadofaragene firadenovec-vncg (brand name Adstiladrin) for adult patients with high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumors.
Efficacy was evaluated in Study CS-003, a multicenter, single-arm trial enrolling 157 patients with high-risk non-muscle invasive bladder cancer, 98 of whom had Bacillus Calmette-Guérin -unresponsive carcinoma in situ evaluable for response. Patients received nadofaragene firadenovec-vncg 75 mL intravesical instillation (3 x 1011 viral particles/mL) once every three months up to 12 months, unacceptable toxicity, or recurrent high-grade non-muscle invasive bladder cancer. Patients without high-grade recurrence were allowed to continue nadofaragene firadenovec-vncg every three months.
The major efficacy outcome measures were complete response at any time and duration of response. Complete response was defined as negative cystoscopy with applicable transurethral resection of bladder tumor and biopsies and urine cytology. Random bladder biopsies of five sites were conducted in patients remaining in complete response at 12 months. The complete response rate was 51%, the median duration of response was 9.7 months, and 46% of responding patients remained in complete response for at least one year.
The most common adverse events (with an incidence of 10% or more), including laboratory abnormalities (greater than 15%), were increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition urgency, increased creatinine, hematuria, decreased phosphate, chills, dysuria, and pyrexia.
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
Full prescribing information for these approvals can be found on the web at www.fda.gov/, key word search Approved Cellular and Gene Therapy Products.
Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at MedWatch: The FDA Safety Information and Adverse Event Reporting Program.
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