13 August 2018 - The approval of a new drug to treat polyneuropathy caused by a rare and frequently fatal disease called hereditary transthyretin-mediated amyloidosis marks the arrival of a game-changing new class of therapeutics. 

The new drug, called patisiran (Onpattro), is a small interfering RNA that is part of a class of therapeutics that can target hereditary diseases by affecting gene function. 

With this ability to target specific RNA “messages,” we now have the potential to design therapeutics for a wide range of diseases. Patisiran is the first drug approved to treat hereditary transthyretin-mediated amyloidosis.

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