Decoding Regulatory Agency Decisions: The Case of Amylyx’s Relyvrio/Albrioza In The US And EU

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

US EU FLAGS — EU and US regulators took different approaches to regulatory flexibility when it came to Amylyx's ALS treatment • Source: Shutterstock

Is the risk of delaying patient access to a desperately needed potential treatment greater than the risk of facilitating access to an ineffective but relatively safe product? How important is regulatory precedence? And how much should patients influence the final outcome?

Key Takeaways

Amylyx’s Relyvrio/Albrozia for treating amyotrophic lateral sclerosis was refused approval in the EU but was approved in the US before it was withdrawn following poor Phase III study results.

These are some of the issues that the US Food and Drug Administration took into consideration when it decided whether...