<unk> , sold under the brand name Onpattro, is a medication for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis, a fatal rare disease that is estimated to affect 50,000 people worldwide. 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. It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.<unk>  utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.<unk>  was developed and is marketed by Alnylam. The FDA considers it to be a first-in-class medication.
Patisiran
Tezacaftor
Enarodustat
Influenzanet
Bepridil
Zileuton
Fasudil
Belinostat