By Chris Wack

Regenxbio said Tuesday that the U.S. Food and Drug Administration has granted a regenerative medicine advanced therapy designation for RGX-121, an investigational one-time AAV therapeutic for the treatment of mucopolysaccharidosis Type II, also known as MPS II or Hunter syndrome.

RMAT designation is designed to expedite the drug development and review processes for promising new treatments, including gene therapies. The designation recognizes that the preliminary clinical evidence from RGX-121 demonstrates its potential to address unmet medical needs for MPS II.

Regenxbio said RGX-121 is currently being studied in a trial that is enrolling MPS II patients. The company continues to support plans to file a biologics license application for RGX-121 in 2024 using the accelerated approval pathway.

A drug is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and if preliminary clinical evidence indicates the drug or therapy has the potential to address unmet medical needs for such disease or condition.

A Phase I/II trial of RGX-121 for the treatment of MPS II in pediatric patients over the age of five years old is ongoing.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

May 23, 2023 07:40 ET (11:40 GMT)

Copyright (c) 2023 Dow Jones & Company, Inc.
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