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Biotech · FDA Approval · Gene Therapy · Rare Disease
The U.S. FDA reversed its previous decision, indicating that existing data for Regenxbio's rare-disease gene therapy, Navsunli, for Hunter syndrome, supports an accelerated approval application, leading to a 16% surge in Regenxbio shares on Monday.
This decision marks the latest in a series of regulatory reversals, following similar shifts for uniQure's Huntington's gene therapy and Replimune's skin cancer drug. Barclays analyst Eliana Merle stated this signals increased FDA flexibility, suggesting the agency is "more friendly to industry moving forward" despite leadership changes.
The FDA's feedback is a significant turnaround for Regenxbio, which previously faced uncertainty over Navsunli's trial design. Raymond James analyst Sean McCutcheon affirmed this development signals "a new approach which is significantly more flexible and portends a favorable outcome for Regenxbio and patients with Hunter Syndrome." The FDA has confirmed Regenxbio will not need to enroll additional patients or conduct new studies, including a previously requested placebo-controlled trial.
Regenxbio plans to meet with the agency in July and expects to resubmit its application in the third quarter for expedited review.