The drug is the second ever made by using a Nobel prize-winning technique called RNA interference, which allows scientists to mute genes that aren’t functioning properly. It is designed to treat acute hepatic porphyria, which causes attacks of severe abdominal pain and can cause permanent neurological damage and death.
Givlaari is priced at $575,000 per year, though Alnylam has said that after discounts it expects the price to be $442,000 per year, according to STAT. Each vial of the drug costs $39,000.
The price of the drug is subject to agreements with insurers that link the actual cost to how well patients respond to it. Additionally, if an insurance provider covers more patients than expected, Alnylam will pay rebates to the insurer, a deal that has never been done before for rare disease drugs, according to STAT.
Alnylam expects 3,000 patients in the U.S. and Europe to be eligible for the drug. Despite the small number, analysts projected peak sales as high as $600 million.
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