This section highlights FDA-related milestones and regulatory updates for drugs developed by MeiraGTx (MGTX).
Over the past two years, MeiraGTx has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AAV2-hAQP1, AAV-GAD, and rAAV8.hRKp.AIPL1. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
AAV2-hAQP1 - FDA Regulatory Timeline and Events
AAV2-hAQP1 is a drug developed by MeiraGTx for the following indication: For the Treatment of Grade 2/3 Radiation-Induced Xerostomia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AAV2-hAQP1
- Announced Date:
- December 9, 2024
- Indication:
- For the Treatment of Grade 2/3 Radiation-Induced Xerostomia
Announcement
MeiraGTx 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).
AI Summary
MeiraGTx announced that the FDA has granted RMAT designation to its gene therapy, AAV2-hAQP1, for the treatment of Grade 2/3 radiation-induced xerostomia (RIX). This designation highlights the promising preliminary clinical data showing that AAV2-hAQP1 may offer significant relief for patients suffering from debilitating dry mouth after radiation treatment for head and neck cancers.
The RMAT designation, granted under the 21st Century Cures Act, gives MeiraGTx benefits similar to those of Fast Track and Breakthrough Therapy designations. These benefits include more frequent interactions with the FDA, potential accelerated approval, and priority review. The decision reflects the FDA’s confidence in the early clinical results and the therapy’s ability to address a critical unmet medical need, paving the way for faster development and review of this promising treatment option.
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AAV-GAD - FDA Regulatory Timeline and Events
AAV-GAD is a drug developed by MeiraGTx for the following indication: for the treatment of Parkinson's disease, MGT-GAD-025.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- AAV-GAD
- Announced Date:
- May 9, 2025
- Indication:
- for the treatment of Parkinson's disease, MGT-GAD-025.
Announcement
MeiraGTx Holdings plc announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the treatment of Parkinson's disease not adequately controlled with anti-Parkinsonian medications.
AI Summary
MeiraGTx Holdings plc announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its gene therapy, AAV-GAD, for treating Parkinson’s disease that is not adequately controlled with current anti-Parkinsonian medications. This designation reflects positive results from three clinical studies, including a dose-escalation Phase 1 study, a double-blind Phase 2 study, and a randomized dose-ranging study. The studies showed significant improvements in motor symptoms as measured by the UPDRS Part 3 score and other validated assessments. RMAT status provides benefits such as accelerated development, enhanced interaction with the FDA, and potential accelerated approval and Priority Review. MeiraGTx is optimistic that this one-time stereotactic infusion treatment, which targets a key area in the brain, has the potential to modify the progression of Parkinson’s disease and improve patient outcomes.
Read Announcement- Drug:
- AAV-GAD
- Announced Date:
- March 13, 2025
- Indication:
- for the treatment of Parkinson's disease, MGT-GAD-025.
Announcement
MeiraGTx Holdings plc announced a broad strategic collaboration with Hologen Limited, a world-leading developer of multi-modal generative AI foundation models of real-world clinical data for clinical medicine and pharmaceutical drug development.
AI Summary
MeiraGTx Holdings plc announced a strategic collaboration with Hologen Limited, a global leader in multi-modal generative AI models built on real-world clinical data. The two companies are forming a joint venture, Hologen Neuro AI Ltd, through which Hologen will provide its cutting-edge AI capabilities to transform clinical medicine and drug development. The focus of the collaboration is to fully fund and accelerate the development of AAV-GAD for Parkinson’s disease and explore other therapies targeting the central nervous system.
Under this agreement, MeiraGTx will receive $200 million upfront while Hologen commits up to an additional $230 million for the joint venture. MeiraGTx will lead the clinical development and manufacturing efforts, using Hologen’s AI technology to optimize drug discovery and manufacturing processes. This partnership represents a significant step forward in leveraging AI to improve treatment outcomes for neurological disorders.
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rAAV8.hRKp.AIPL1 - FDA Regulatory Timeline and Events
rAAV8.hRKp.AIPL1 is a drug developed by MeiraGTx for the following indication: For the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- rAAV8.hRKp.AIPL1
- Announced Date:
- February 21, 2025
- Indication:
- For the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy
Announcement
MeiraGTx Holdings plc announced the publication of results from the first-in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy.
AI Summary
MeiraGTx Holdings plc announced the publication of results from their first-in-human study treating children with AIPL1-associated severe retinal dystrophy. The study, published in The Lancet, involved young children who were legally blind at birth due to this inherited condition. In the initial cohort, four children received a one-time unilateral subretinal injection of rAAV8.hRKp.AIPL1, showing improved visual acuity, functional vision, and evidence of protection against further retinal degeneration.
Building on these promising results, seven additional children were treated bilaterally with the same gene therapy. All 11 children demonstrated meaningful benefits from the treatment, indicating that early gene supplementation can be both safe and effective. This breakthrough study offers new hope for addressing one of the most severe forms of inherited blindness and marks a significant step forward in pediatric gene therapy.
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