FDA pauses Regenxbio gene therapy trial again due to safety concerns

Live updates

1. 20d ago

   FDA halts Regenxbio Hunter syndrome gene therapy trial over safety concerns

   The FDA has placed a clinical hold on Regenxbio's gene therapy for Hunter syndrome, citing safety concerns. This action halts testing and disrupts the company's plans to resubmit its application. The hold stems from findings related to spine MRI scans. The news coincided with a crash in RGNX stock prices.

   Why it matters

   Hunter syndrome is a rare genetic disorder requiring specialized treatment. This is not the first time the FDA has paused this specific trial. The resubmission process is now stymied by the current regulatory hold.

   What is confirmed

   • The FDA placed a clinical hold on Regenxbio's gene therapy for Hunter syndrome.
   • The FDA's decision was driven by safety concerns.
   • The hold has disrupted Regenxbio's plans to resubmit its application.

   Still unconfirmed

   • RGNX stock crashed following the news.

   What to watch next

   • Regenxbio's response to the spine MRI findings
   • FDA decision on lifting the clinical hold
   • Updated timelines for the therapy resubmission
   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. Reuters — FDA places clinical hold on Regenxbio's gene therapy for Hunter Syndrome
   7. pharma.economictimes.indiatimes.com — FDA places clinical hold on Regenxbio's gene therapy for Hunter Syndrome
   confidence 90%