Patisiran: First Global Approval

@article{Hoy2018PatisiranFG,
  title={Patisiran: First Global Approval},
  author={Sheridan M. Hoy},
  journal={Drugs},
  year={2018},
  volume={78},
  pages={1625-1631}
}
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… 
View on Springer
ncbi.nlm.nih.gov
228 Citations
Highly Influential Citations
3
Background Citations
56
Methods Citations
5

228 Citations

Citation Type

Citation Type

The growth of siRNA-based therapeutics: updated clinical studies.
Toward the therapeutic application of small interfering RNA bioconjugates in the central nervous system
Ionizable liposomal siRNA therapeutics enables potent and persistent treatment of Hepatitis B
TLDR
This study provides an effective nucleic acid delivery system and a promising RNAi agent for the treatment of hepatitis B and the toxicity profiles suggested satisfactory safety outcomes with ten times the therapeutic window.
Oligonucleotide Therapeutics: From Discovery and Development to Patentability
TLDR
This review provides an overview of therapeutic oligonucleotides, in particular the different types of oligon nucleotides and their modes of action, the tissues they target and the routes by which they are administered to patients, and the therapeutic areas inWhich they are used.
The nanomedicine rush: New strategies for unmet medical needs based on innovative nano DDS.
Plasma and Liver Protein Binding of N-Acetylgalactosamine–Conjugated Small Interfering RNA
TLDR
An orthogonally validated 50 kDa molecular-mass cutoff ultrafiltration assay was developed and a qualitative screen to identify binding “hits” across a panel of select purified human plasma proteins was developed to gain insight into which specific plasma proteins bind to siRNA.
The infinite possibilities of RNA therapeutics
TLDR
General aspects of many of the molecules currently used in RNA therapeutics, including in vitro transcribed mRNA, antisense oligonucleotides (ASOs), aptamers, small interfering RNAs (siRNAs), and microRNAs (miRNAs) are presented.
Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach
TLDR
The concept of SNALPs is reviewed, presenting a series of examples on their efficacy as siRNA nanodelivery systems, and hopes to unveil the therapeutic potential of these nanosystems for targeted brain delivery of siRNAs in LSDs.
Opportunities and challenges for antisense oligonucleotide therapies
TLDR
An overview of the different AON approaches is provided, with a focus on AONs developed for inborn errors of metabolism, as well as therapeutic options for inherited metabolic diseases as well.
Nanoparticle Delivery Platforms for RNAi Therapeutics Targeting COVID-19 Disease in the Respiratory Tract
TLDR
Advances in this field have the potential for antiviral treatments of COVID-19 disease and could be adapted to treat a range of respiratory diseases.
...
...

References

SHOWING 1-10 OF 13 REFERENCES
Safety and efficacy of RNAi therapy for transthyretin amyloidosis.
BACKGROUND Transthyretin amyloidosis is caused by the deposition of hepatocyte-derived transthyretin amyloid in peripheral nerves and the heart. A therapeutic approach mediated by RNA interference…
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
TLDR
Patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis with polyneuropathy and showed an effect on gait speed and modified BMI.
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
TLDR
Patisiran was generally well tolerated and resulted in significant dose-dependent knockdown of transthyretin protein in patients with FAP, and is currently in phase III development.
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis: Analysis of the APOLLO Study
TLDR
Patisiran decreased mean left ventricular wall thickness, global longitudinal strain, N-terminal prohormone of brain natriuretic peptide, and adverse cardiac outcomes compared with placebo at month 18, suggesting that patisIRan may halt or reverse the progression of the cardiac manifestations of hATTR amyloidosis.
Evolving landscape in the management of transthyretin amyloidosis
TLDR
The TTR stabilizers tafamidis and diflunisal slow disease progression in some patients with ATTR amyloidosis with polyneuropathy, and the postulated synergistic effect of doxycycline and tauroursodeoxycholic acid on dissolution of amyloids is under investigation.
PHASE 2 OPEN-LABEL EXTENSION (OLE) STUDY OF PATISIRAN, AN INVESTIGATIONAL RNA INTERFERENCE (RNAI) THERAPEUTIC FOR THE TREATMENT OF HEREDITARY ATTR AMYLOIDOSIS WITH POLYNEUROPATHY
EFFECT OF PATISIRAN ON NERVE FIBER DENSITY AND AMYLOID CONTENT IN SKIN : RESULTS FROM PHASE 2 OPEN LABEL EXTENSION (OLE) STUDY IN HATTR AMYLOIDOSIS
TLDR
Results from phase 2 of the open label extension study in HATTR AMYLOIDOSIS showed positive results for phosphorous concentration and yin andyang levels.
On Medicine
What were to happen if we were to unravel the concept of ‘medicine’ – and with that, writing? And with that, philosophy? In ‘On Medicine’, Kélina Gotman journeys through philosophical concepts of…
Onpattro 2 mg/mL concentrate for solution for infusion: EU summary of product characteristics. 2018
  • http://www.ema.europ a.eu/ema/. Accessed
  • 2018
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease [media release
  • 2018
...
...