The biotech firm intends to refile its application for approval during the third quarter. This move follows a scheduled formal meeting with the FDA in July aimed at resolving ongoing disputes over the therapy's clinical pathway. The agency previously denied the application, but the latest developments suggest a path forward for the treatment of this aggressive genetic disorder.
In section Market Quotes
Regenxbio Surges as FDA Reverses Course on Rare Disease Therapy
Regenxbio shares climbed 12% to $8.75 in premarket trading following reports that the Food and Drug Administration will reconsider its rejection of the company’s experimental gene therapy. The shift follows a regulatory impasse regarding Navsunli, a treatment intended for patients suffering from the fatal, rare condition known as Hunter syndrome.
Navsunli addresses Hunter syndrome, a condition characterized by irreversible brain damage and cell death, which typically claims the lives of patients by their midteens. The disease remains extremely rare, impacting approximately 2,000 individuals globally—predominantly boys—with roughly 50 new cases diagnosed annually within the United States.
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