The IRA’s single-orphan conundrum

Alnylam’s announcement that it was pausing and reconsidering plans to develop Amvuttra to treat Stargardt disease illuminated a conundrum embedded in the Inflation Reduction Act. It provides an exemption from price-setting for drugs that are approved for a single orphan condition — but that exemption disappears if the drug is approved for any other indication. 

Approval for Stargardt could cause Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) to lose the exemption, which Amvuttra vutrisiran is eligible for as a result of its approval to treat polyneuropathy associated with hereditary TTR-mediated amyloidosis (ATTR-PN) in adults, an orphan indication. ...