Regenxbio reported on Monday that the FDA issued a complete response letter regarding the biologics license application for its treatment, known as RGX-121. The agency’s decision focused on whether the clinical study properly identified appropriate patient groups and if the historical data used for comparison was accurate. Furthermore, regulators questioned whether the biological markers measured in spinal fluid were strong enough to predict tangible health benefits for those suffering from the condition.
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FDA Rejects Regenxbio Gene Therapy for Rare Hunter Syndrome
The U.S. Food and Drug Administration has declined to approve Regenxbio’s gene therapy for Hunter syndrome, a rare and fatal neurodegenerative disease, citing concerns over trial data and patient selection.
Data Challenges and Resubmission Plans
The FDA had previously accepted the application under its accelerated approval pathway in May 2025. However, the current rejection stalls the rollout of a therapy aimed at an ultra-rare disease that currently lacks effective treatments for irreversible neurological decline. CEO Curran Simpson expressed disappointment with the feedback, stating that the company remains confident in the quality and volume of evidence supporting the long-term potential of the therapy.Regenxbio intends to meet with regulators to establish a path forward and resubmit its application as quickly as possible. The company emphasized that the data collected so far demonstrates a positive trajectory for patients with Hunter syndrome, a condition that requires urgent intervention to prevent permanent damage.
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