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Alnylam has achieved multiple FDA and EMA approvals for first-in-class RNAi products (ONPATTRO/patisiran for hATTR amyloidosis, GIVLAARI/givosiran for acute hepatic porphyria, OXLUMO/lumasiran for primary hyperoxaluria type 1, AMVUTTRA/vutrisiran for ATTR amyloidosis polyneuropathy and cardiomyopathy, Qfitlia/fitusiran for hemophilia A and B), with positive late-stage results for zilebesiran (hypertension). These drugs demonstrate robust target protein knockdown, durable clinical benefit, and favorable safety profiles in their indications[2][6][12][16].
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