FDA Denies Regenxbio Therapy in Latest Rare Disease Setback (1)

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.

Regenxbio shares fell as much as 15% in premarket trading in New York on Tuesday. The stock had fallen 28% this year through Monday’s close, a decline sparked by the company saying last month that a related trial had ...