The United States Food and Drug Administration has awarded Fast Track Designation to an experimental gene therapy for the treatment of wet age-related macular degeneration. For more detailed information, you can view the source press release at Adverum Biotechnologies’ website by clicking here.
About Age-Related Macular Degeneration (AMD)
AMD is a condition that affects a person’s vision, particularly in the middle. Typically, it first appears around the ages of fifty to sixty. It doesn’t usually lead to complete blindness, however, it may make some everyday activities more difficult, such as reading. If AMD is left untreated, it can progress and cause vision to worsen. If this happens slowly over several years it is known as ‘dry AMD’, while if it occurs very quickly (over weeks or months) it is often called ‘wet AMD’, or wAMD. According to the source article, approximately one in ten people with AMD will have the wAMD type. This information was sourced from the NHS, and you can read more about it here.
About Adverum’s Gene Therapy
The investigational gene therapy ADVM-022 is being researched as a potential treatment for wAMD. Current treatments for wAMD may need to be frequently re-administered, which may be challenging for some patients. However, a lack of treatment can cause further vision loss. The experimental gene therapy is designed to reduce the burden of frequent treatments for patients.
Preclinical Research into the Gene Therapy
A preclinical study supported the safety and efficacy of ADVM-022. A single injection of the therapy was found to be more effective at preventing Grade IV lesions than no treatment. The therapy was also well tolerated, and no serious adverse events were reported.
Plans for a Clinical Trial
Following these promising pre-clinical results, Aderum has announced its plans to carry out a Phase 1 clinical trial of the gene therapy. It will be a dose-escalation study designed to evaluate the safety and tolerability of ADVM-022 in approximately eighteen people who have wAMD. The study will be held across several centres in the US, and people who take part will be followed for two years.
Fast Track Designation
Recently, the US FDA has awarded ADVM-022 Fast Track Designation for the treatment of wAMD. This status is designed to help get new drugs to patients more quickly. Drugs and developers with Fast Track Designation may be eligible for more frequent meetings with the FDA, more FDA communication, and a faster review process. For more information about this designation, click here.