RNAi Technologies and Global Markets (BIO127C) |
Onpattro (patisiran) |
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FDA approved patisiran in 2018, for the treatment of the polyneuropathy of hereditary, transthyretin-mediated amyloidosis or hATTR amyloidosis in adults. |
Givlaari (givosiran) |
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FDA approved givosiran in 2019, for the treatment of adults with acute hepatic porphyria, or AHP. In EU it is approved for the treatment of AHP in adults and adolescents aged 12 years and older. |
Oxlumo (lumasiran) |
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FDA approved lumasiran in 2020, for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. |
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