12:30 PM EDT, 06/18/2024 (MT Newswires) -- Regenxbio ( RGNX ) said Tuesday it has completed a "successful" pre-biologics license application meeting with the US Food and Drug Administration for RGX-121 for the treatment of Mucopolysaccharidosis Type II, or Hunter Syndrome.

"This positive engagement with the FDA marks an important milestone for Regenxbio ( RGNX ) and RGX-121 on the path towards potential approval of the first gene therapy for Hunter syndrome," said COO Curran Simpson.

The company said it expects to initiate submission of a rolling application in Q3 and an FDA inspection of its Manufacturing Innovation Center in the first half of 2025. It also expects a confirmatory trial to begin in the second half of 2025.

"Based on an expected priority review, potential approval of the planned BLA could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025," the company said.

Shares of Regenxbio ( RGNX ) were up about 3% in recent trading.

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