BioCentury
ARTICLE | Politics, Policy & Law

IRA prompts Alnylam to reconsider rare indication

Law’s orphan exemption leads company to pause trial for Stargardt disease

October 28, 2022 9:00 PM UTC

The potential for the Inflation Reduction Act to hurt patients has, until now, been theoretical. It became real Wednesday for the 30,000 Americans diagnosed with Stargardt disease when Alnylam announced that the law had prompted it to pause and reconsider plans to start a Phase III trial of vutrisiran for the disease.

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) is the first company to publicly announce that it may abandon plans to add an indication to an orphan drug label, but it won’t be the last. The Stargardt situation illustrates the difficult decisions drug companies will be forced to make, as well as the consequences for patients...