View source for Biology:Nadofaragene firadenovec

{{Short description|Gene therapy medication}}
{{Infobox drug
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<!-- Gene therapy data -->
| gt_target_gene       = Interferon alfa-2b
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<!-- Clinical data -->
| pronounce         = 
| tradename         = Adstiladrin
| Drugs.com         = 
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| licence_CA        = <!-- Health Canada may use generic or brand name (generic name preferred) -->
| licence_EU        = <!-- EMA uses INN (or special INN_EMA) -->
| DailyMedID        = Nadofaragene firadenovec
| licence_US        = <!-- FDA may use generic or brand name (generic name preferred) -->
| pregnancy_AU      = <!-- A / B1 / B2 / B3 / C / D / X -->
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| routes_of_administration = Intravesical
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| ATC_prefix        = None
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| legal_US          = Rx-only
| legal_US_comment  = <ref name="Adstiladrin FDA label">{{Cite web |url=https://www.fda.gov/media/164029/download |title=Archived copy |website=[[Organization:Food and Drug Administration|Food and Drug Administration]] |access-date=17 December 2022 |archive-date=17 December 2022 |archive-url=https://web.archive.org/web/20221217055100/https://www.fda.gov/media/164029/download |url-status=live }}</ref><ref>{{cite web | title=Adstiladrin | publisher=U.S. [[Organization:Food and Drug Administration|Food and Drug Administration]] (FDA) | date=16 December 2022 | url=https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/adstiladrin | access-date=16 December 2022 | id=STN: BLA 125700 | archive-date=17 December 2022 | archive-url=https://web.archive.org/web/20221217055104/https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/adstiladrin | url-status=live }}</ref>
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| UNII              = 0OOS09O1FH
| KEGG              = D12531
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| synonyms          = Nadofaragene firadenovec-vncg, Instilidrin, rAd-IFN/Syn3

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}}

'''Nadofaragene firadenovec''', sold under the brand name '''Adstiladrin''', is a [[Biology:Gene therapy|gene therapy]] for the treatment of [[Medicine:Bladder cancer|bladder cancer]].<ref name="Adstiladrin FDA label" /><ref name="FDA PR 20221216" /><ref name="FDA Adstiladrin" /> It is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy.<ref name="FDA PR 20221216">{{cite press release | title=FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer | publisher=U.S. [[Organization:Food and Drug Administration|Food and Drug Administration]] (FDA) | date=16 December 2022 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer | access-date=16 December 2022 | archive-date=16 December 2022 | archive-url=https://web.archive.org/web/20221216214436/https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer | url-status=live }} {{PD-notice}}</ref>

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.<ref name="FDA Adstiladrin" />

Nadofaragene firadenovec was approved for medical use in the United States in December 2022.<ref name="FDA PR 20221216" /><ref name="FDA Adstiladrin">{{cite web | title=FDA approves first adenoviral vector-based gene therapy for high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer | website=U.S. [[Organization:Food and Drug Administration|Food and Drug Administration]] (FDA) | date=19 December 2022 | url=https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-first-adenoviral-vector-based-gene-therapy-high-risk-bacillus-calmette-guerin | access-date=19 December 2022 | archive-date=19 December 2022 | archive-url=https://web.archive.org/web/20221219203951/https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-first-adenoviral-vector-based-gene-therapy-high-risk-bacillus-calmette-guerin | url-status=live }} {{PD-notice}}</ref><ref>{{cite press release | title=Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer | website=Ferring Pharmaceuticals USA | date=16 December 2022 | url=https://ferringusa.com/?press=ferring-receives-approval-from-u-s-fda-for-adstiladrin-for-high-risk-bcg-unresponsive-non-muscle-invasive-bladder-cancer | access-date=16 December 2022 | archive-date=17 December 2022 | archive-url=https://web.archive.org/web/20221217074455/https://ferringusa.com/?press=ferring-receives-approval-from-u-s-fda-for-adstiladrin-for-high-risk-bcg-unresponsive-non-muscle-invasive-bladder-cancer | url-status=live }}</ref>

== 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.<ref name="Adstiladrin FDA label" /><ref name="FDA PR 20221216" />

== 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.<ref name="FDA PR 20221216" /><ref name="FDA Adstiladrin" /> 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.<ref name="FDA PR 20221216" /><ref name="FDA Adstiladrin" /> 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).<ref name="FDA PR 20221216" /> The median duration of response was 9.7 months.<ref name="FDA PR 20221216" /> Forty-six percent of responding participants remained in complete response for at least one year.<ref name="FDA PR 20221216" /> The major efficacy outcome measures were complete response at any time and duration of response.<ref name="FDA Adstiladrin" /> Complete response was defined as negative cystoscopy with applicable transurethral resection of bladder tumor and biopsies and urine cytology.<ref name="FDA Adstiladrin" /> Random bladder biopsies of five sites were conducted in participants remaining in complete response at twelve months.<ref name="FDA Adstiladrin" />

The US [[Organization:Food and Drug Administration|Food and Drug Administration]] (FDA) granted the application for nadofaragene firadenovec [[Medicine:Priority review|priority review]], [[Medicine:Breakthrough therapy|breakthrough therapy]], fast track, and [[Chemistry:Orphan drug|orphan drug]] designations.<ref name="FDA PR 20221216" /><ref name="FDA Adstiladrin" /> The FDA granted approval of Adstiladrin to Ferring Pharmaceuticals A/S.<ref name="FDA PR 20221216" />

== Society and culture ==
=== Names ===
Nadofaragene firadenovec is the [[Medicine:International nonproprietary name|international nonproprietary name]] (INN).<ref name=WHORecList79>{{cite journal | vauthors = ((World Health Organization)) | title = International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 79 | journal = WHO Drug Information | volume = 32 | issue = 1 | year = 2018 | hdl = 10665/330941 | hdl-access = free | }}</ref>

== References ==
{{reflist}}

== External links ==
* {{ClinicalTrialsGov|NCT02773849|Adstiladrin  (Instilidrin) in Patients With High Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)}}


{{DEFAULTSORT:Nadofaragene firadenovec}}
[[Category:Breakthrough therapy]]
[[Category:Cancer treatments]]
[[Category:Gene therapy]]
[[Category:Orphan drugs]]



{{Sourceattribution|Nadofaragene firadenovec|1}}