The IRA’s single-orphan conundrum
The IRA’s orphan exemption could force manufacturers of at least 18 drugs to reconsider plans to add indications
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. ...