MeiraGTx Holdings plc (MGTX) Receives RMAT Designation from FDA for AAV2-hAQP1

On December 9, 2024, MeiraGTx Holdings plc announced 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). This designation acknowledges the promising clinical evidence of the potential benefits of AAV2-hAQP1 as a one-time therapy for this condition.

MeiraGTx’s President and CEO, Alexandria Forbes, Ph.D., expressed enthusiasm for the RMAT designation, recognizing its significance in potentially enhancing the lives of patients grappling with xerostomia post-radiation treatment. The designation highlights the advanced regenerative nature of the gene therapy, its targeting of a serious condition, and the demonstrated clinical evidence indicating its ability to address an unmet medical need.

Established under the 21st Century Cures Act, the RMAT designation facilitates the development and review of Regenerative Medicine therapeutic candidates, including human gene therapies, for severe or life-threatening diseases. This designation enables closer collaboration with the FDA, expedited discussions on product development, and potential acceleration of the approval process.

MeiraGTx’s AAV2-hAQP1 had previously received Orphan Drug Designation by the FDA. The Phase 1 AQUAx clinical trial demonstrated significant improvements in patient-reported outcomes and saliva production without any treatment-related adverse events. The ongoing Phase 2 AQUAx2 study is enrolling participants in multiple locations to further assess the therapy’s efficacy.

Grade 2/3 radiation-induced xerostomia is a debilitating consequence affecting a notable percentage of patients treated with head and neck cancer radiation. AAV2-hAQP1, administered locally to the salivary gland through a minimally invasive procedure, presents a potentially transformative treatment option for this unmet medical need.

MeiraGTx (NASDAQ: MGTX) is a clinical-stage genetic medicines company with a robust pipeline of late-stage programs. The company’s focus on genetic medicine aims to address unmet medical needs and expand treatment options for various diseases.

For more information about MeiraGTx and its programs, please visit the company’s official website.

This press release contains forward-looking statements reflecting management’s current expectations and should be considered in conjunction with risk factors outlined in MeiraGTx’s SEC filings. The company disclaims any obligation to update these forward-looking statements based on subsequent events or changes in circumstances.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read MeiraGTx’s 8K filing here.

MeiraGTx Company Profile

MeiraGTx Holdings plc, a clinical stage gene therapy company, focusing on developing treatments for patients with serious diseases. The company develops various therapies for ocular diseases, including inherited retinal diseases and large degenerative ocular diseases, neurodegenerative diseases, and xerostomia.