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

Eplontersen, 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]

//////////////

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

join me on Linkedin

Anthony Melvin Crasto Ph.D – India | LinkedIn

join me on Researchgate

RESEARCHGATE

join me on Facebook

Anthony Melvin Crasto Dr. | Facebook

• Twitter
• FACEBOOK

join me on twitter

Anthony Melvin Crasto Dr. | twitter

+919321316780 call whatsaapp

EMAIL. amcrasto@amcrasto

//////////

References

1. ^ Jump up to:^a b c d https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217388s000lbl.pdf
2. ^ “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.
3. ^ 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.
4. ^ 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.
5. ^ 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.
6. ^ “Eplontersen: FDA-Approved Drugs”. U.S. Food and Drug Administration (FDA). Retrieved 21 December 2023.
7. ^ “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.
8. ^ “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.
9. ^ 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