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patisiran (Onpattro)
Indications:
- treatment of hereditary transthyretin amyloidosis
- treatment of polyneuropathy due to transthyretin amyloidosis [3]
* benefit continues for 5 years [6]
- disability, polyneuropathy, & quality of life modestly improved [6]
Dosage:
- 0.3 mg/kg every 3 weeks
5 ML patisiran lipid complex 2 MG/ML injection
Mechanism of action:
- small interfering RNA
- inhibits hepatic synthesis of transthyretin
Notes:
- orphan drug
- cost estimate: $345,000-$450,000/year
General
short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA)
neurologic agent
References
- Adams DA, Gonzalez-Duarte A, O'Riordan WD et al
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin
Amyloidosis.
N Engl J Med 2018; 379:11-21. July 5.
PMID: 29972753
https://www.nejm.org/doi/full/10.1056/NEJMoa1716153
- Yasgur BS
New Agents Show Promise for Severe and Fatal Genetic Disease.
Medscape - Jul 10, 2018.
https://www.medscape.com/viewarticle/899112
- Brooks M
FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR.
Medscape - Aug 10, 2018.
https://www.medscape.com/viewarticle/900541
- Wikipedia: Patisiran
https://en.wikipedia.org/wiki/Patisiran
- RxNorm
- George J
Patisiran Shows Long-Term Benefit in Hereditary ATTR With Polyneuropathy.
RNAi drug led to modest changes in disability, severity, and quality of
life over 5 years.
MedPage Today January 15, 2025
https://www.medpagetoday.com/neurology/generalneurology/113787
- Adams D, Wixner J, Polydefkis M et al
Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis
With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.
JAMA Neurol. 2025 Jan 13.
PMID: 39804640
https://jamanetwork.com/journals/jamaneurology/fullarticle/2828606