patisiran (Onpattro)
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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
More general terms
- 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: https://www.ncbi.nlm.nih.gov/pubmed/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
- ↑ 3.0 3.1 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
- ↑ 6.0 6.1 6.2 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: https://www.ncbi.nlm.nih.gov/pubmed/39804640 https://jamanetwork.com/journals/jamaneurology/fullarticle/2828606