Eplontersen

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Eplontersen

AKCEA-TTR-LRx

ION-682884 FREE ACID
ISIS-682884 FREE ACID

UNII0GRZ0F5XJ6

CAS number1637600-16-8

ALL-P-AMBO-5-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXOXY))PHOSPHO)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)GUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)ADENYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE

FDA APP, 12/21/2023, To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis, Wainua

AKCEA-TTR-LRx is under investigation in clinical trial NCT04136184 (Neuro-ttransform: A Study to Evaluate the Efficacy and Safety of Akcea-ttr-lrx in Participants With Hereditary Transthyretin-mediated Amyloid Polyneuropathy).

Eplontersen, sold under the brand name Wainua, is a medication used for the treatment of transthyretin-mediated amyloidosis.^[1] It is a transthyretin-directed antisense oligonucleotide.^[1] It was developed to treat hereditary transthyretin amyloidosis by Ionis Pharmaceuticals and AstraZeneca.^[2]^[3]^[4]^[5]

It was approved for medical use in the United States in December 2023.^[6]^[7]^[8]

Medical uses

Eplontersen is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.^[1]

Society and culture

Names

Eplontersen is the international nonproprietary name.^[9]

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Eplontersen
Clinical data
Trade names	Wainua
Other names	AKCEA-TTR-LRx
AHFS/Drugs.com	Eplontersen
License data	US DailyMed: Eplontersen
Routes of administration	Subcutaneous
ATC code	N07XX21 (WHO)
Legal status
Legal status	US: ℞-only^[1]
Identifiers
CAS Number	1637600-16-8
DrugBank	DB16199
UNII	0GRZ0F5XJ6

References

^ Jump up to:^a ^b ^c ^d https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217388s000lbl.pdf
^ “Ionis announces FDA acceptance of New Drug Application for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)” (Press release). Ionis Pharmaceuticals. 7 March 2023. Archived from the original on 26 September 2023. Retrieved 21 December 2023 – via PR Newswire.
^ Coelho, Teresa; Waddington Cruz, Márcia; Chao, Chi-Chao; Parman, Yeşim; Wixner, Jonas; Weiler, Markus; et al. (February 2023). “Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen”. Neurology and Therapy. 12 (1): 267–287. doi:10.1007/s40120-022-00414-z. PMC 9837340. PMID 36525140.
^ Coelho, Teresa; Marques, Wilson; Dasgupta, Noel R.; Chao, Chi-Chao; Parman, Yeşim; França, Marcondes Cavalcante; et al. (October 2023). “Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy”. The Journal of the American Medical Association. 330 (15): 1448–1458. doi:10.1001/jama.2023.18688. PMC 10540057. PMID 37768671.
^ Diep, John K.; Yu, Rosie Z.; Viney, Nicholas J.; Schneider, Eugene; Guo, Shuling; Henry, Scott; et al. (December 2022). “Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis”. British Journal of Clinical Pharmacology. 88 (12): 5389–5398. doi:10.1111/bcp.15468. PMID 35869634. S2CID 250989659.
^ “Eplontersen: FDA-Approved Drugs”. U.S. Food and Drug Administration (FDA). Retrieved 21 December 2023.
^ “Wainua (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis”. Ionis Pharmaceuticals, Inc. (Press release). 21 December 2023. Retrieved 22 December 2023.
^ “Wainua (eplontersen) granted first-ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis”. AstraZeneca US (Press release). 22 December 2023. Retrieved 22 December 2023.
^ World Health Organization (2021). “International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85”. WHO Drug Information. 35 (1). hdl:10665/340684.

External links

Clinical trial number NCT04136184 for “NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy” at ClinicalTrials.gov
Clinical trial number NCT01737398 for “Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy” at ClinicalTrials.gov

///////////Eplontersen, Wainua, FDA 2023, APPROVALS 2023, ION-682884 FREE ACID, ISIS-682884 FREE ACID

It's only fair to share...

Eplontersen

Eplontersen

Medical uses

Society and culture

Names

AS ON DEC2021 3,491,869 VIEWS ON BLOG WORLDREACH AVAILABLEFOR YOUR ADVERTISEMENT

Anthony Melvin Crasto Ph.D – India | LinkedIn

RESEARCHGATE

Anthony Melvin Crasto Dr. | Facebook

References

External links

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