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6h ago

FDA Places Clinical Hold on Regenxbio’s RGX-121 After Expanded MRI Finds Spinal Masses

Regulators now require more imaging to determine whether the small, asymptomatic spine findings are linked to the therapy, delaying any near-term U.S. approval filing.

Signage is seen outside of the Food and Drug Administration headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly//File Photo
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Overview

  • The U.S. Food and Drug Administration put RGX-121 on clinical hold on Monday after an expanded MRI monitoring program detected small nodules or cystic masses on the spines of five treated patients.
  • All five patients received RGX-121 via intracisternal or intraventricular delivery three to six years ago and remain without symptoms while showing stability or improvement on neurocognitive assessments.
  • Investigators and radiologists classified the spine findings as non-serious and likely benign but said there is no pathological evidence tying them to the gene therapy and causality remains unresolved.
  • Regenxbio said it will pause near-term resubmission of its Biologics License Application, will review additional imaging with partner NS Pharma, and plans longer-term follow-up to address FDA questions.
  • The action hit markets hard with Regenxbio shares tumbling about 25% and a brief trading halt, and it adds to heightened regulatory scrutiny of AAV-based gene therapies after a related RGX-111 program was linked to a brain tumor.