June 22 (Reuters) - Regenxbio said on Monday that the U.S. FDA had indicated it would reverse the earlier rejection of the company's rare-disease gene therapy, and that existing data would be sufficient to support an accelerated approval application.
Shares of the Rockville, Maryland-based drug developer were up 10% in premarket trading.
In February, the U.S. Food and Drug Administration declined to approve the gene therapy, Navsunli, for Hunter syndrome, citing uncertainty over the trial design.
Hunter
syndrome is a rare genetic disorder in which the body is unable to break down certain sugar molecules, affecting physical and cognitive development.
The move reflects a broader FDA shift on rare-disease therapies and signals an effort to mend ties with the sector, after acting FDA Commissioner Kyle Diamantas met with advocacy groups earlier this month.
Regenxbio said it plans to seek a formal meeting with the agency in July and aims to resubmit its application in the third quarter.
The company also said the FDA indicated it would not need to enroll additional patients or conduct new studies, including a previously requested placebo-controlled trial.
The FDA said it would review Regenxbio's resubmission on an expedited basis, with labeling discussions expected to begin shortly after the refiling.
Last week, uniQure said the FDA reversed course on its gene therapy for Huntington's, clearing the path for an accelerated approval filing after previously calling for a new trial.
Separately, Replimune said in May it planned to seek approval for its experimental skin cancer drug for a third time after reaching an agreement with the U.S. regulator.
The Wall Street Journal reported about the development earlier on Monday.
(Reporting by Siddhi Mahatole in Bengaluru; Editing by Diti Pujara)
