FDA Approvals

In another reversal for rare disease field, FDA to reconsider Regenxbio gene therapy

FierceBiotech Jun 23, 2026 FDA Setback

In another sign that the FDA is easing its stance toward rare disease drug candidates under new leadership, the agency will reconsider Regenxbio’s gene therapy for an ultra-rare childhood disease, a reversal coming just about four months after a rejection.

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