Dive Brief:
The Food and Drug Administration has placed another clinical hold on a Regenxbio gene therapy after study investigators detected abnormalities on the MRIs of five trial participants.
The company said Monday that masses that are “likely” benign were found in patients who received the treatment, RGX-121, about three to six years ago for a condition known as Hunter syndrome. Those patients did not exhibit any symptoms and “continue to do well clinically and have demonstrated overall stability to improvement on neurocognitive and neurobehavioral assessments,” Regenxbio said.
Still, the announcement represents the latest regulatory setback for RGX-121 this year. The FDA put testing on pause in January due to other safety worries and rejected Regenxbio’s application a month later. Regenxbio had been preparing a new submission after aligning with regulators, but now no longer plans to file that application in “the near term,” the company said Monday. Shares plunged more than 25% in early Monday trading.
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