Biology:Nadofaragene firadenovec
| Gene therapy | |
|---|---|
| Target gene | Interferon alfa-2b |
| Clinical data | |
| Trade names | Adstiladrin |
| Other names | Nadofaragene firadenovec-vncg, Instilidrin, rAd-IFN/Syn3 |
| License data | |
| Routes of administration | Intravesical |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
| UNII | |
| KEGG | |
Nadofaragene firadenovec, sold under the brand name Adstiladrin, is a gene therapy for the treatment of bladder cancer.[1][3][4] It is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy.[3]
The most common adverse events including laboratory abnormalities, include increased glucose, instillation site discharge, increased triglycerides, fatigue, bladder spasm, micturition urgency, increased creatinine, hematuria, decreased phosphate, chills, dysuria, and pyrexia.[4]
Nadofaragene firadenovec was approved for medical use in the United States in December 2022.[3][4][5]
Medical uses
Nadofaragene firadenovec is indicated for the treatment of adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.[1][3]
History
The safety and effectiveness of nadofaragene firadenovec was evaluated in a multicenter clinical study (Study CS-003 (NCT02773849)) that included 157 participants with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer, 98 of whom had BCG-unresponsive carcinoma in situ with or without papillary tumors and could be evaluated for response.[3][4] Participants received nadofaragene firadenovec once every three months for up to twelve months, or until unacceptable toxicity to therapy or recurrent high-grade non-muscle-invasive bladder cancer.[3][4] Overall, 51% of enrolled participants using nadofaragene firadenovec therapy achieved a complete response (the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine).[3] The median duration of response was 9.7 months.[3] Forty-six percent of responding participants remained in complete response for at least one year.[3] The major efficacy outcome measures were complete response at any time and duration of response.[4] Complete response was defined as negative cystoscopy with applicable transurethral resection of bladder tumor and biopsies and urine cytology.[4] Random bladder biopsies of five sites were conducted in participants remaining in complete response at twelve months.[4]
The US Food and Drug Administration (FDA) granted the application for nadofaragene firadenovec priority review, breakthrough therapy, fast track, and orphan drug designations.[3][4] The FDA granted approval of Adstiladrin to Ferring Pharmaceuticals A/S.[3]
Society and culture
Names
Nadofaragene firadenovec is the international nonproprietary name (INN).[6]
References
- ↑ 1.0 1.1 1.2 "Archived copy". https://www.fda.gov/media/164029/download.
- ↑ "Adstiladrin". U.S. Food and Drug Administration (FDA). 16 December 2022. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/adstiladrin.
- ↑ 3.00 3.01 3.02 3.03 3.04 3.05 3.06 3.07 3.08 3.09 3.10 "FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer" (Press release). U.S. Food and Drug Administration (FDA). 16 December 2022. Archived from the original on 16 December 2022. Retrieved 16 December 2022.
This article incorporates text from this source, which is in the public domain.
- ↑ 4.0 4.1 4.2 4.3 4.4 4.5 4.6 4.7 4.8 "FDA approves first adenoviral vector-based gene therapy for high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer". 19 December 2022. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-first-adenoviral-vector-based-gene-therapy-high-risk-bacillus-calmette-guerin. This article incorporates text from this source, which is in the public domain.
- ↑ "Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer". Ferring Pharmaceuticals USA (Press release). 16 December 2022. Archived from the original on 17 December 2022. Retrieved 16 December 2022.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 79". WHO Drug Information 32 (1). 2018.
External links
- Clinical trial number NCT02773849 for "Adstiladrin (Instilidrin) in Patients With High Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)" at ClinicalTrials.gov
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