There are no available data on WAINUA use in pregnant women to inform drug-associated risk of adverse developmental outcomes. Eplontersen treatment leads to a decrease in serum vitamin A levels and vitamin A supplementation is advised for patients taking eplontersen. Vitamin A is essential for normal embryofetal development; however, excessive vitamin A levels are associated with adverse developmental effects. The effect of vitamin A supplementation on the fetus in the setting of a reduction in maternal serum TTR caused by eplontersen administration is unknown.L49479
No adverse developmental effects were observed when eplontersen or a mouse-specific surrogate was administered to mice prior to mating and continuing throughout organogenesis.L49479
No dose adjustment is required in patients ?65 years of age. In Study NCT04136184, 44 (31%) patients were 65 to 74 years of age, and 8 (5.6%) patients were ?75 years of age. No overall differences in safety or effectiveness were observed between these patients and younger adult patients, but a greater sensitivity of some older individuals cannot be ruled out.L49479
Carcinogenicity studies have not been conducted with eplontersen.L49479
Eplontersen was negative for genotoxicity in in vitro (bacterial reverse mutation, chromosomal aberration in Chinese hamster lung cells) and in vivo (mouse bone marrow micronucleus) assays.L49479
Subcutaneous administration of eplontersen (0, 5, 25, or 75 mg/kg) or a mouse-specific surrogate (25 mg/kg) to male and female mice weekly prior to and during mating and continuing in females throughout the period of organogenesis resulted in no adverse
effects on fertility.L49479
Eplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl galactosamine residues for hepatic delivery. It was previously investigated as a potential treatment for hereditary transthyretin-mediated amyloidosis.A262975 Hereditary transthyretin-mediated amyloidosis is caused primarily by pathogenic variants of the TTR gene, leading to the formation and thus accumulation of insoluble and misfolded amyloid in multiple organs, although wild-type misfolded TTR has also been observed to contribute to the disease pathophysiology.A262985 As eplontersen targets both the WT and mutant TTR, it poses tremendous promises as a treatment.A262990
On December 21, 2023, the FDA approved eplontersen under the brand name WAINUA for the treatment of adult polyneuropathy of hereditary transthyretin-mediated amyloidosis. This approval was based on positive results demonstrated in the 35-week interim analysis from the Phase 3 NEURO-TTRansform study, where a consistent improvement in the Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) Score were observed.L49525
Fitur visualisasi ini dikembangkan menggunakan pendekatan Graph Theory untuk memetakan hubungan polifarmasi dan molekuler. Entitas (Obat, Target, Gen) direpresentasikan sebagai Simpul (Nodes), sedangkan hubungan biologisnya sebagai Sisi (Edges).
drugbank-id dan name pada skema XML DrugBank.targets/target yang memuat polipeptida sasaran.gene-name dan varian snp-effects.Tata letak grafik menggunakan algoritma Force-Directed Graph (Barnes-Hut). Model fisika ini menerapkan gaya tolak-menolak antar simpul (Gravitasi: -3000) agar tidak tumpang tindih, serta gaya pegas (Spring: 0.04) pada garis penghubung untuk fleksibilitas interaksi.