MEDWEBUS00113 v20 August 2021 Approved Gene Therapies ADA adenosine deaminase CD cluster of differentiation cDNA complementary DNA EC European Commission FDA US Food and Drug Administration NMPA National Medical Products Administration formerly the China Food and Drug Administrati ID: 909564
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Slide1
Gene Therapy Approvals
2021
MEDWEB-US-00113 v2.0 August 2021
Slide2Approved Gene Therapies
ADA, adenosine deaminase; CD, cluster of differentiation; cDNA, complementary DNA; EC, European Commission; FDA, U.S. Food and Drug Administration; NMPA, National Medical Products Administration (formerly the China Food and Drug Administration); SCID, severe combined immunodeficiency.
1.
EMA.
Strimvelis. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis. Accessed May 24, 2021; 2. U.S. FDA. News release. August 30, 2017. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-tisagenlecleucel-b-cell-all-and-tocilizumab-cytokine-release-syndrome. Accessed May 24, 2021; 3. U.S. FDA. News release. May 1, 2018. Available at: https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm606540.htm. Accessed May 24, 2021; 4. EMA. Kymriah. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/kymriah. Accessed May 24, 2021; 5. U.S. FDA. News release. October 18, 2017. Available at: https://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm581296.htm. Accessed May 24, 2021; 6. EMA. Yescarta. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/yescarta. Accessed May 24, 2021; 7. EMA. Zynteglo. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/zynteglo. Accessed May 24, 2021; 8. U.S. FDA. News release. July 24, 2020. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-adult-patients-relapsed-or-refractory-mcl. Accessed May 24, 2021; 9. EMA. Tecartus. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/tecartus. Accessed May 24, 2021; 10. EMA. Libmeldy. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy. Accessed May 24, 2021; 11. EC. Libmeldy. Available at: https://ec.europa.eu/health/documents/community-register/html/h1493.htm. Accessed May 27, 2021; 12. U.S. FDA. New release. February 5, 2021. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-adults-relapsed-or-refractory-large-b-cell-lymphoma. Accessed May 24, 2021; 13. U.S. FDA. New release. March 27, 2021. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-first-cell-based-gene-therapy-adult-patients-multiple-myeloma. Accessed May 25, 2021; 14. Bluebird Bio. Press release. July 21, 2021. Available at: https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-ec-approval-skysonatm-elivaldogene#. Accessed July 23, 2021.
2
Gene therapies have been approved for the treatment of certain cancers and inherited diseases, including1–13:
Agent
Disease Area
Approval date
US (FDA)
EU (EC)
China (NMPA)
Ex vivo
treatments
A
utologous CD34+ cells encoding the human ADA cDNA sequence
1
Severe combined
immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)
–
May 2016
–
Tisagenlecleucel
2–4
B-cell precursor acute lymphoblastic leukemia; large B-cell lymphoma
August 2017;
May 2018
August 2018
–
Axicabtagene
ciloleucel
5,6
Large B-cell lymphoma
October 2017
August 2018
–
Betibeglogene
autotemcel
7
Transfusion-dependent β-thalassemia without β
0
/β
0
genotype
–
May 2019
–
Brexucabtagene
autoleucel
8,9
Mantle cell lymphoma
July 2020
December 2020
–
Atidarsagene
autotemcel
10,11
Metachromatic leukodystrophy
–
December 2020
–
Lisocabtagene
maraleucel
12
Large B-cell lymphoma
February 2021
–
–
Idecabtagene
vicleucel
13
Multiple myeloma
March 2021
_
_
Elivaldogene
autotemcel
14
Cerebral adrenoleukodystrophy
_
July 2021
_
Slide3Approved Gene Therapies
*Alipogene tiparvovec (brand name:
Glybera
®
) was approved by the EC in October 2012 for the treatment of familial lipoprotein lipase deficiency. However, due to extremely limited usage, it is no longer marketed17; †Indicates date of authorization for emergency use.EC, European Commission; FDA, U.S. Food and Drug Administration; NMPA, National Medical Products Administration (formerly the China Food and Drug Administration); RPE65, retinal pigment epithelium-specific 65 kDa protein. 1. BioPharm International. Available at: http://www.biopharminternational.com/genesis-gendicine-story-behind-first-gene-therapy. Accessed July 9, 2021; 2. Amgen. News release. October 27, 2015. Available at: https://www.amgen.com/media/news-releases/2015/10/fda-approves-imlygic-talimogene-laherparepvec-as-first-oncolytic-viral-therapy-in-the-us/. Accessed May 24, 2021; 3. Amgen. News release. December 17, 2015. Available at: https://www.amgen.com/media/news-releases/2015/12/european-commission-approves-amgens-imlygic-talimogene-laherparepvec-as-first-oncolytic-immunotherapy-in-europe/. Accessed May 24, 2021; 4. U.S. FDA. News release. December 18, 2017. Available at: https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm589467.htm. Accessed July 9, 2021; 5. Luxturna® [product information]. Available at: https://www.ema.europa.eu/en/documents/product-information/luxturna-epar-product-information_en.pdf. Accessed June 22, 2021; 6. U.S. FDA. News release. May 24, 2019. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease. Accessed July 9, 2021; 7.
EMA. Zolgensma. Available at: https://www.ema.europa.eu
/en/medicines/human/EPAR/zolgensma. Accessed May 24, 2021; 8.
Zabdeno
®
[product information]. Available at: https://www.ema.europa.eu/en/documents/product-information/zabdeno-epar-product-information_en.pdf. Accessed July 9, 2021; 9. EMA. Zabdeno. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/zabdeno. Accessed July 9, 2021; 10. U.S. FDA. Pfizer-BioNTech COVID-19 Vaccine. Available at: https://www.fda.gov/emergency-preparedness-and-response/coronavirus-disease-2019-covid-19/pfizer-biontech-covid-19-vaccine. Accessed July 9, 2021; 11. EMA. Comirnaty Assessment Report. Available at: https://www.ema.europa.eu/en/documents/assessment-report/comirnaty-epar-public-assessment-report_en.pdf. Accessed July 13, 2021; 12. U.S. FDA. Moderna COVID-19 Vaccine. Available at: https://www.fda.gov/emergency-preparedness-and-response/coronavirus-disease-2019-covid-19/moderna-covid-19-vaccine. Accessed July 9, 2021; 13. Spikevax® [product information]. Available at: https://www.ema.europa.eu/en/documents/product-information/spikevax-previously-covid-19-vaccine-moderna-epar-product-information_en.pdf. Accessed July 9, 2021; 14. Vaxzevria® [product information]. Available at: https://www.ema.europa.eu/en/documents/product-information/vaxzevria-previously-covid-19-vaccine-astrazeneca-epar-product-information_en.pdf. Accessed July 9, 2021; 15. U.S. FDA. Janssen COVID-19 Vaccine. Available at: https://www.fda.gov/emergency-preparedness-and-response/coronavirus-disease-2019-covid-19/janssen-covid-19-vaccine. Accessed July 9, 2021; 16. COVID-19 Vaccine Janssen [product information]. Available at: https://www.ema.europa.eu/en/documents/product-information/covid-19-vaccine-janssen-epar-product-information_en.pdf. Accessed July 9, 2021; 17. UniQure. Press release. April 20, 2017. Available at: http://uniqure.com/GL_PR_Glybera%20withdrawal_FINAL_PDF.pdf. Accessed July 13, 2021.
3
Gene therapies have been approved for the treatment of certain cancers, and inherited and infectious diseases, including1–17:
Agent
Disease Area
Approval date
US (FDA)
EU (EC)
China (NMPA)
In vivo
treatments*
Gendicine
1
Head and neck squamous cell carcinoma
–
–
October 2003
Talimogene laherparepvec
2,3
Metastatic melanoma
October 2015
December 2015
Voretigene
neparvovec
4,5
Biallelic
RPE65
mutation-associated retinal dystrophy
December 2017
November 2018
–
Onasemnogene
abeparvovec
6,7
Spinal muscular atrophy
May 2019
May 2020
–
Ebola vaccine (Ad26.ZEBOV-GP)
8,9
Active immunization for prevention of disease caused by the Ebola virus
-
July 2020
-
Covid-19 mRNA vaccine (BNT162b2)
10,11
Active immunization to prevent COVID-19 caused by SARS-CoV-2
December 2020
†
December 2020
†
-
Covid-19 mRNA vaccine (mRNA 1273)
12,13
December 2020
†
January 2021
†
-
Covid-19 vaccine (ChAdOx1-S)
14
-
January 2021
†
-
Covid-19 vaccine (Ad26.COV2-S)
15,16
Active immunization to prevent COVID-19 caused by SARS-CoV-2
February 2021
†
March 2021
†
-