Publication Cover
Amyloid
The Journal of Protein Folding Disorders
Volume 30, 2023 - Issue 1
31,708
Views
28
CrossRef citations to date
0
Altmetric
Research Articles

Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

ORCID Icon, , ORCID Icon, , ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, ORCID Icon, , ORCID Icon, , , ORCID Icon & show all
Pages 18-26 | Received 28 Feb 2022, Accepted 16 Jun 2022, Published online: 23 Jul 2022

References

  • Hawkins PN, Ando Y, Dispenzeri A, et al. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625–638.
  • Mohty D, Damy T, Cosnay P, et al. Cardiac amyloidosis: updates in diagnosis and management. Arch Cardiovasc Dis. 2013;106(10):528–540.
  • Hanna M. Novel drugs targeting transthyretin amyloidosis. Curr Heart Fail Rep. 2014;11(1):50–57.
  • Adams D, Coelho T, Obici L, et al. Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study. Neurology. 2015;85(8):675–682.
  • Kelly JW. Amyloid fibril formation and protein misassembly: a structural quest for insights into amyloid and prion diseases. Structure. 1997;5(5):595–600.
  • Klimtchuk ES, Prokaeva T, Frame NM, et al. Unusual duplication mutation in a surface loop of human transthyretin leads to an aggressive drug-resistant amyloid disease. Proc Natl Acad Sci USA. 2018;115:E6428–E6436.
  • Koike H, Katsuno M. Ultrastructure in transthyretin amyloidosis: from pathophysiology to therapeutic insights. Biomedicines. 2019;7(1):11.
  • Mangione PP, Verona G, Corazza A, et al. Plasminogen activation triggers transthyretin amyloidogenesis in vitro. J Biol Chem. 2018;293(37):14192–14199.
  • Shin SC, Robinson-Papp J. Amyloid neuropathies. Mt Sinai J Med. 2012;79(6):733–748.
  • Conceição I, Gonzalez-Duarte A, Obici L, et al. "Red-flag" symptom clusters in transthyretin familial amyloid polyneuropathy. J Peripher Nerv Syst. 2016;21(1):5–9.
  • Adams D, Koike H, Slama M, et al. Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease. Nat Rev Neurol. 2019;15(7):387–404.
  • Gertz MA. Hereditary ATTR amyloidosis: burden of illness and diagnostic challenges. Am J Manag Care. 2017;23(7 Suppl):S107–S112.
  • Adams D, Suhr OB, Hund E, et al. First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy. Curr Opin Neurol. 2016;29(Suppl. 1):S14–S26.
  • Swiecicki PL, Zhen DB, Mauermann ML, et al. Hereditary ATTR amyloidosis: a single-institution experience with 266 patients. Amyloid. 2015;22(2):123–131.
  • Sattianayagam PT, Hahn AF, Whelan CJ, et al. Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant. Eur Heart J. 2012;33(9):1120–1127.
  • Gertz MA, Kyle RA, Thibodeau SN. Familial amyloidosis: a study of 52 North American-born patients examined during a 30-year period. Mayo Clin Proc. 1992;67(5):428–440.
  • Castaño A, Drachman BM, Judge D, et al. Natural history and therapy of TTR-cardiac amyloidosis: emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs. Heart Fail Rev. 2015;20(2):163–178.
  • Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA. 2013;310(24):2658–2667.
  • Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012;79(8):785–792.
  • Adams D, Gonzalez-Duarte A, O'Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):11–21.
  • Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):22–31.
  • Alnylam Pharmaceuticals Inc. US prescribing information: AMVUTTRA (vutrisiran) injection, for subcutaneous use. 2022. [cited June 28, 2022]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215515s000lbl.pdf.
  • Habtemariam BA, Karsten V, Attarwala H, et al. Single-dose pharmacokinetics and pharmacodynamics of transthyretin targeting N-acetylgalactosamine-small interfering ribonucleic acid conjugate, vutrisiran, in healthy subjects. Clin Pharmacol Ther. 2021;109(2):372–382.
  • Soprano DR, Herbert J, Soprano KJ, et al. Demonstration of transthyretin mRNA in the brain and other extrahepatic tissues in the rat. J Biol Chem. 1985;260(21):11793–11798.
  • Holmgren G, Steen L, Ekstedt J, et al. Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30). Clin Genet. 1991;40(3):242–246.
  • Adams D, Suhr OB, Dyck PJ, et al. Trial design and rationale for APOLLO, a phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol. 2017;17(1):181.
  • Solomon SD, Adams D, Kristen A, et al. Effects of patisiran, an RNA interference therapeutic, on cardiac parameters in patients with hereditary transthyretin-mediated amyloidosis. Circulation. 2019;139(4):431–443.
  • Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369(9):819–829.
  • Alnylam Pharmaceuticals Inc. US prescribing information: ONPATTRO (patisiran) lipid complex injection, for intravenous use. 2020. [cited February 17, 2022]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/210922s007lbl.pdf.
  • European Medicines Agency. Summary of product characteristics: Onpattro 2 mg/mL concentrate for solution for infusion. 2018. [cited February 17, 2022]. Available from: https://www.ema.europa.eu/documents/product-information/onpattro-epar-product-information_en.pdf.
  • Zhang X, Goel V, Attarwala H, et al. Patisiran pharmacokinetics, pharmacodynamics, and exposure-response analyses in the phase 3 APOLLO trial in patients with hereditary transthyretin-mediated (hATTR) amyloidosis. J Clin Pharmacol. 2020;60(1):37–49.
  • Suanprasert N, Berk JL, Benson MD, et al. Retrospective study of a TTR FAP cohort to modify NIS + 7 for therapeutic trials. J Neurol Sci. 2014;344(1-2):121–128.
  • Jackevicius CA, Mamdani M, Tu JV. Adherence with statin therapy in elderly patients with and without acute coronary syndromes. JAMA. 2002;288(4):462–467.
  • Strauch B, Petrák O, Zelinka T, et al. Precise assessment of noncompliance with the antihypertensive therapy in patients with resistant hypertension using toxicological serum analysis. J Hypertens. 2013;31(12):2455–2461.