MeiraGTx Holdings plc announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1. This designation is for the treatment of Grade 2/3 radiation-induced xerostomia (RIX), a severe and debilitating condition with no current treatment options.
The RMAT designation recognizes the preliminary clinical evidence demonstrating the potential benefit of AAV2-hAQP1 as a one-time treatment. RIX affects approximately 30-40% of patients undergoing radiation treatment for head and neck cancer, representing a significant unmet medical need with over 170,000 patients in the U.S. and an additional 15,000 new patients annually.
The RMAT designation provides several benefits, including those of Fast Track and Breakthrough Therapy designations, such as increased interaction with the FDA, rolling review, and potential Priority Review of a Biologics License Application (BLA). This aims to expedite the development and review process for AAV2-hAQP1, which had previously received Orphan Drug Designation from the FDA.
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