FDA Denies Regenxbio Therapy in Latest Rare Disease Setback

Regenxbio Inc. shares tumbled after US regulators rejected its gene therapy for Hunter syndrome, underscoring the hard line the Trump administration is taking on drug approvals for rare diseases.

The Food and Drug Administration determined that Regenxbio’s clinical trial data wasn’t enough to support approval in the condition, which causes brain damage in boys, the company said in a statement Monday.