Alnylam Blames New US Drug Pricing Laws for Shelving of Rare Eye Disease Therapeutic

October 28, 2022

Alnylam is setting aside a clinical trial of its therapeutic Vutrisiran for a rare eye disease due to the looming US drug pricing reforms, according to the company. The RNA-based treatment for Stargardt disease is currently used in another rare disease but would not face Medicare pricing negotiations because the law only targets newly approved drugs. Approval of the treatment for Stargardt would allow the government to negotiate the price of Vutrisiran for that indication.

According to Phil Taylor, “Alnylam’s earlier plan was to start trials of the drug before the end of this year in the genetic eye disease, which causes fatty material to build up in the macula area of the retina and affect vision. There is currently no approved treatment, although both drug- and gene-based therapies are in development.”

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(Source: PharmaPhorum, October 28th, 2022)

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