Second RNAi-based drug gains FDA approval, targeting rare genetic condition causing severe abdominal pain

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Image: Kris Tripplaar/Sipa USA

The FDA has approved the second-ever drug based on a Nobel prize-winning technique known as RNA interference, Alnylam’s givosiran.

The medicine is intended to treat a rare genetic condition called acute hepatic porphyria that can cause attacks of severe abdominal pain. The drug, which will be branded as Givlaari, will cost $575,000 per year, the company announced [November 20]; after discounts, it expects the average price to be $442,000 per year. Each vial of the drug will cost $39,000.

RNA interference, or RNAi, lets scientists mute genes that aren’t functioning properly. Although scientists pioneered the technique decades ago, the FDA only approved the first RNAi-based drug, another of Alnylam’s, last year.

Related article:  Were ‘crucial contributors’ snubbed in awarding Nobel prize for cancer immunotherapy?

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The drug’s approval is a good sign for other companies developing drugs based on the RNAi technique. The drug is the first to use a type of sugar called GalNAc that allows more precise delivery of drugs to liver cells — which could, potentially, reduce serious side effects associated with the drug going elsewhere in the body.

“This really is an important milestone for the whole field,” [Alnylam CEO John] Maraganore said.

Read full, original post: FDA approves Alnylam’s Givlaari, second-ever drug based on RNAi

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