What's Happening?
The FDA has postponed its decision on Regenxbio's gene therapy for Hunter syndrome, clemidsogene lanparvovec, by three months to review additional clinical data. The therapy aims to deliver a functional copy of the IDS gene to the central nervous system, addressing the genetic mutation causing the disease. Despite the delay, Regenxbio remains on track with its launch preparations, having received positive feedback from a recent FDA inspection.
Why It's Important?
The delay in FDA approval highlights the complexities involved in gene therapy development and regulatory review. Hunter syndrome is a rare genetic disorder, and the approval of clemidsogene lanparvovec could provide a significant breakthrough in treatment options. The therapy's potential to reduce reliance on enzyme replacement therapy represents a major advancement in addressing the disease's underlying causes.
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What's Next?
Regenxbio anticipates a decision by February 8, 2026, and continues its launch preparations. The outcome of the FDA review will be closely watched by stakeholders, as it could influence future gene therapy developments and regulatory processes.
