MeiraGTx Holdings plc (MGTX), a vertically integrated, clinical stage genetic medicines company, announced Monday that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1 for the treatment of Grade 2/3 radiation-induced xerostomia (RIX).
The RMAT designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for this debilitating condition.
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising Regenerative Medicine therapeutic candidates, including human gene therapies that treat, modify, reverse or cure serious or life-threatening diseases.
Similar to Breakthrough Therapy designation, RMAT designation allows for increased interaction with the FDA and immediate multidisciplinary comprehensive discussions of the ongoing product development program, clinical trials and plans for expediting the manufacturing development strategy.
RMAT designation includes the benefits of Fast Track and Breakthrough Therapy designations with rolling review and potential Priority Review of a product's biologics license application (BLA). It also allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review
AAV2-hAQP1 was previously granted Orphan Drug Designation by the FDA.
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