Patisiran
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Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis,[7] a fatal rare disease that is estimated to affect 50,000 people worldwide.[8][9]
Clinical data | |
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Trade names | Onpattro |
Other names | ALN-18328 |
AHFS/Drugs.com | Monograph |
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Routes of administration | Intravenous |
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Formula | C412H520N148O290P40 |
Molar mass | 13424.388 g·mol−1 |
It is the first small interfering RNA-based drug approved by the U.S. Food and Drug Administration (FDA) and the first drug approved by the FDA to treat this condition.[8] It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.[10] Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[11]
Patisiran was developed and is marketed by Alnylam.[12] The FDA considers it to be a first-in-class medication.[13]