Patisiran: First Global Approval

Patisiran (ONPATTRO™) is a double-stranded small interfering RNA encapsulated in a lipid nanoparticle for delivery to hepatocytes. By specifically binding to a genetically conserved sequence in the 3′ untranslated region of mutant and wild-type transthyretin (TTR) messenger RNA, patisiran causes its...

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Bibliographic Details
Published in:Drugs (New York, N.Y.) N.Y.), 2018-10, Vol.78 (15), p.1625-1631
Main Author: Hoy, Sheridan M.
Format: Article
Language:English
Subjects:
3' Untranslated regions
AdisInsight Report
Adults
Amyloidosis
Conserved sequence
Disease
Double-stranded RNA
Drug Approval
FDA approval
Genetic Therapy
Hepatocytes
Histamine
Humans
Infusions, Intravenous
Internal Medicine
Intravenous administration
Intravenous infusion
Lipids
Medicine
Medicine & Public Health
mRNA
Mutation
Nanoparticles
Nanoparticles - chemistry
Patients
Pharmaceuticals
Pharmacology/Toxicology
Pharmacotherapy
Polyneuropathies - genetics
Polyneuropathies - therapy
Polyneuropathy
Prealbumin - genetics
Proteins
Ribonucleic acid
RNA
RNA, Small Interfering - administration & dosage
RNA, Small Interfering - chemistry
RNA-mediated interference
siRNA
Transthyretin
United States
United States Food and Drug Administration
Weighing
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Description
Summary:Patisiran (ONPATTRO™) is a double-stranded small interfering RNA encapsulated in a lipid nanoparticle for delivery to hepatocytes. By specifically binding to a genetically conserved sequence in the 3′ untranslated region of mutant and wild-type transthyretin (TTR) messenger RNA, patisiran causes its degradation (via RNA interference) and subsequently a reduction in serum TTR protein levels and tissue TTR protein deposits. Patisiran has been developed by Alnylam Pharmaceuticals; it was recently approved in the USA for the treatment of the polyneuropathy of hereditary TTR-mediated amyloidosis (hATTR) in adults and subsequently approved in the EU for the treatment of hATTR in adults with stage 1 or 2 polyneuropathy. The recommended dosage, administered as a single intravenous infusion over approximately 80 min, is 0.3 mg/kg once every 3 weeks for patients weighing
ISSN:0012-6667
1179-1950
DOI:10.1007/s40265-018-0983-6