FDA Approves Denali's Hunter Syndrome Drug, Signaling Shift in Rare Disease Policy

The FDA's approval of Denali's Avlayah, despite recent stricter policies, highlights its confidence in innovative therapies for rare diseases. This move may influence future regulatory decisions and industry strategies. Stakeholders are watching for how this approval impacts ongoing drug development and approval processes, including the pending decision on off-the-shelf CAR-T therapies by Allogene.