Aug 24 (Reuters) – Regenxbio said on Monday the U.S. Food and Drug Administration has placed its experimental gene therapy for Hunter syndrome on clinical hold after spinal scans found abnormalities in five study participants.
Spinal scans identified a small lump or fluid-filled mass in five patients who received the treatment, RGX-121, about three to six years ago, the biotech said. Its shares were halted in premarket trading.
Hunter syndrome, also known as MPS II, is a rare inherited disorder that can cause progressive damage to the brain and other organs and tissues.
Regenxbio said it does not expect to resubmit its application for approval of the treatment in the near term.
(Reporting by Padmanabhan Ananthan and Kunal Das in Bengaluru; Editing by Shilpi Majumdar)
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