Patients & Families
Inherited Retinal Disease
We currently have four ongoing clinical stage programs in inherited retinal diseases (IRDs), along with a compassionate use program for those with Leber congenital amaurosis (LCA) due to mutations in the AIPL1 gene. IRDs are a group of rare eye disorders caused by an inherited gene mutation that can result in individuals experiencing severe vision loss, impairment or blindness. Our four ongoing clinical stage programs include: X-Linked Retinitis Pigmentosa (XLRP), Achromatopsia (ACHM) due to mutations both in in the CNGB3 and CNGA3 genes, and RPE65-deficiency.
MeiraGTx Clinical Trials
Learn more about MeiraGTx’s ocular programs and determine if you may be eligible to participate in one of our IRD gene therapy clinical trials or ongoing natural history studies:
To learn more about our compassionate use program for those with LCA due to mutations in the AIPL1 gene, please email patients@meiragtx.com.
IRD Patient Resources
To find out more about genetic testing for IRDs, please visit the My Retina Tracker website provided by the Foundation Fighting Blindness.
For additional information about IRDs, please visit the following organizations to find resources and support:
MeiraGTx is focused on developing potentially curative treatments for patients living with serious diseases. Our goal is to provide access to our product candidates at the appropriate time and in the correct manner for patients.
Expanded access refers to the use of a product candidate outside the auspices of a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient for which no alternative treatments are available. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. Expanded access use is usually requested by physicians on behalf of their patients for whom participating in a clinical trial is difficult or impracticable. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
A number of factors consistent with the policies of the U.S. Food and Drug Administration (FDA) and guidelines of international regulatory agencies should be taken into account when considering expanded access. They include:
- The illness must be serious or life-threatening
- There are no other viable treatment options (such as approved products or enrolling clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks based on safety and efficacy information available at the time
- Ability to provide a product in a fair and equitable manner, so that there is adequate manufacturing capacity for ongoing programs
- Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay the conduct of current clinical trials or regulatory submissions designed to make the product candidate available to many more patients
At this time, MeiraGTx believes that participation in one of our clinical trials is the most appropriate way to access our product candidates. If you have additional questions, please speak with your physician or contact patients@meiragtx.com. We anticipate acknowledging receipt of requests sent to this e-mail within five business days.
Pursuant to the 21st Century Cures Act, MeiraGTx may revise this policy at any time.
Dated: February 2021
