Patisiran: First Global Approval

Drugs

Volumn 78, Issue 15, 2018, Pages 1625-1631

  Hoy, Sheridan M ^a  

^a SPRINGER   (New Zealand)

Author keywords

[No Author keywords available]

Indexed keywords

NANOPARTICLE; PATISIRAN; SMALL INTERFERING RNA; TRANSTHYRETIN;

CHEMISTRY; DRUG APPROVAL; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; GENETICS; HUMAN; INTRAVENOUS DRUG ADMINISTRATION; MUTATION; POLYNEUROPATHY; UNITED STATES;

DRUG APPROVAL; GENETIC THERAPY; HUMANS; INFUSIONS, INTRAVENOUS; MUTATION; NANOPARTICLES; POLYNEUROPATHIES; PREALBUMIN; RNA, SMALL INTERFERING; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 85053825555     PISSN: 00126667     EISSN: 11791950     Source Type: Journal    
DOI: 10.1007/s40265-018-0983-6     Document Type: Article

References (17)

1. US National Library of Medicine. Genetics home reference: transthyretin amyloidosis. 2018. https://ghr.nlm.nih.gov/. Accessed 16 Aug 2018
2. Hawkins PN, Ando Y, Dispenzeri A, et al. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625–38
3. US FDA. FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease [media release]. 2018. http://www.fda.gov/
4. Alnylam Pharmaceuticals Inc. Onpattro (patisiran) lipid complex injection, for intravenous use: US prescribing information. 2018. http://www.fda.gov/. Accessed 16 Aug 2018
5. Alnylam Netherlands B.V. Onpattro 2 mg/mL concentrate for solution for infusion: EU summary of product characteristics. 2018. http://www.ema.europa.eu/ema/. Accessed 5 Sept 2018
6. Alnylam Pharmaceuticals Inc. Alnylam receives approval of ONPATTRO™ (patisiran) in Europe [media release]. 2018. http://www.alnylam.com/
7. Alnylam Pharmaceuticals Inc. Alnylam Pharmaceuticals reports first quarter 2018 financial results and highlights recent period activity [media release]. 3 May 2018. http://www.alnylam.com/
8. Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369(9):819–29
9. Suhr OB, Coelho T, Buades J, et al. Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study. Orphanet J Rare Dis. 2015;10:109
10. Adams D, Coelho T, Conceicao I, et al. Phase 2 open-label extension (OLE) study of patisiran, an investigational RNA interference (RNAi) therapeutic for the treatment of hereditary ATTR amyloidosis with polyneuropathy [abstract no. PSY18 plus poster]. Value Health. 2017;20(5):A211–2
11. 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
12. Goel V, Gosselin N, Jomphe C, et al. Population pharmacokinetic (PK)/pharmacodynamic (PD) model of serum transthyretin (TTR) following patisiran-LNP administration in healthy volunteers and patients with hereditary TTR-mediated (hATTR) amyloidosis with polyneuropathy [abstract no. EPR1139 plus presentation]. Eur J Neurol. 2018;25(Suppl. 2):354
13. Polydefkis M, Ebenezer G, Adams D, et al. Effect of patisiran on nerve fiber density and amyloid content in skin: results from phase 2 open label extension (OLE) study in hATTR amyloidosis [abstract no. O3_3]. J Peripher Nerv Syst. 2017;22(3):360
14. Zhang X, Goel V, Attarwala H, et al. Patisiran-LNP pharmacokinetics (PK), pharmacodynamics (PD), and exposure–response (E–R) relationship in patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy [abstract no. EPR2142 plus presentation]. Eur J Neurol. 2018;25(Suppl. 2):460
15. 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: an analysis of the APOLLO study. Circulation. 2018. 10.1161/CIRCULATIONAHA.118.035831
16. Yamashita T, Sekijima Y, Koike H, et al. Patisiran, an investigational RNAi therapeutic for patients with hereditary transthyretin-mediated (hATTR) amyloidosis: phase 3 APOLLO study sub-analysis of Japanese patients [poster PC053]. In: XVIth International Symposium on Amyloidosis (ISA). 2018
17. Suhr O, Gonzalez-Duarte A, O’Riordan W, et al. Long-term use of patisiran, an investigational RNAi therapeutic, in patients with hereditary transthyretin-mediated amyloidosis: baseline demographics and interim data from global open label extension study [poster]. In: XVIth International Symposium on Amyloidosis (ISA). 2018