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Alnylam scraps trial in rare eye disorder, blaming drug price reforms

October 28, 2022

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The IRA was signed by Biden in August and introduced a framework that will require Medicare, the US’s government-run insurance programme for older Americans, to negotiate with manufacturers a “reasonable” price for selected brand name drugs on the basis of factors like safety and effectiveness compared to other drugs and development costs.

At the heart of Alnylam’s concerns is a provision in the legislation that exempts orphan drugs for rare diseases from negotiations – but only if they have only one approved orphan use. Vutrisiran is already approved as Amvuttra to treat rare disease hereditary ATTR amyloidosis, so approval in Stargardt disease would prompt the pricing negotiation mechanism.

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