Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings

Live updates

1. 21d ago

   FDA holds Regenxbio gene therapy trial over spine MRI findings

   The FDA has placed a clinical hold on Regenxbio's gene therapy trial for Hunter syndrome due to asymptomatic spine MRI findings in five participants. This development has derailed the company's plans to resubmit its gene therapy for approval. The hold was imposed after the detection of these findings in the CAMPSIITE study.

   Why it matters

   Regenxbio's gene therapy, RGX-121, is being investigated as a treatment for Hunter syndrome, a rare genetic disorder. The FDA's clinical hold is a significant setback for the company, which had planned to resubmit its application for approval. The safety concerns raised by the FDA are being addressed by Regenxbio.

   What is confirmed

   • The FDA placed a clinical hold on Regenxbio's RGX-121 gene therapy for MPS II.
   • Asymptomatic spine MRI findings were detected in five participants in the CAMPSIITE study.
   • REGENXBIO stock plunged 25% following the FDA's clinical hold.

   What to watch next

   • Regenxbio's response to the FDA's concerns and the timing of the trial's resumption
   • The outcome of the FDA's review of the spine MRI findings and their impact on the gene therapy's approval prospects
   Sources used for this update (7)

   1. Fierce Biotech — Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings
   2. BioPharma Dive — Safety concerns spur FDA to again halt testing of Regenxbio gene therapy
   3. STAT — FDA pauses Regenxbio gene therapy trial again due to safety concerns
   4. Yahoo Finance — Why Did RGNX Stock Crash Today?
   5. BioSpace — REGENXBIO’s Hunter syndrome gene therapy hit by FDA hold, stymieing resubmission plans
   6. finance.yahoo.com — RGNX Stock Slumps 25% on FDA Clinical Hold for RGX-121 Gene Therapy
   7. www.yahoo.com — FDA places clinical hold on REGENXBIO’s RGX-121 in MPS II trial
   confidence 100%