patisiran (Onpattro)
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Indications
- treatment of hereditary transthyretin amyloidosis
- treatment of polyneuropathy due to transthyretin amyloidosis[3]
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
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