Patients with diabetic macular edema (DME), a vision-threatening complication of diabetes, may soon have a new treatment option. The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to a gene therapy called 4D-150. This was announced in a press release by 4D Molecular Therapeutics, a biotechnology company specializing in durable, targeted therapies.
DME occurs when fluid accumulates in the macula, the part of the retina responsible for sharp vision. If left untreated, DME can cause significant vision loss.
Current treatments for DME involve frequent injections into the eye, often every six weeks or longer. These injections are time-consuming and invasive. Robert Kim, M.D., chief medical officer at 4D Molecular Therapeutics, explains that many DME patients have other health issues and may struggle to keep up with this demanding treatment schedule, especially since many are younger and still working. He adds that the RMAT designation allows faster development of 4D-150, which could reduce the treatment burden while protecting patients’ vision.
What is RMAT and How Does 4D-150 Work?
The RMAT designation, created under the 21st Century Cures Act, is given to promising new therapies for serious or life-threatening diseases. It helps speed up the FDA review process by encouraging closer communication between developers and regulators. This means patients may access the treatment sooner.
4D-150 works by delivering a gene therapy directly to retinal cells. This therapy enables the cells to produce aflibercept, the active drug in current standard treatments for DME. Unlike regular injections, 4D-150 is given as a single injection in the doctor’s office. The therapy aims to maintain its effect for years, greatly reducing the need for repeated treatments.
The RMAT designation was based on positive results from the ongoing SPECTRA clinical trial. Dr. Kim says that after 32 weeks, patients showed reduced fluid in the retina and improved vision. Additionally, patients receiving the therapy needed 86% fewer injections compared to standard care. The treatment was also well tolerated, with no eye inflammation reported.
Expert Opinions on 4D-150
John W. Kitchens, M.D., an ophthalmologist not involved in the study, acknowledges that current anti-VEGF therapies, like aflibercept, effectively improve vision for many DME patients. These treatments can restore vision in 30% to 40% of cases and reduce the severity of diabetic retinopathy, another serious diabetes complication.
However, anti-VEGF treatments require frequent injections because the benefits wear off over time. Dr. Kitchens says the idea of a single injection lasting for years is very promising. He also notes that fewer injections would ease the workload on clinics and improve patient compliance.
Looking Ahead
4D-150 has also received RMAT designation for wet age-related macular degeneration (wet AMD), another serious eye disease. It is the first investigational therapy to receive this status for both conditions.
Dr. Kitchens highlights that Phase 2 trials for wet AMD showed 80% of patients experienced a large reduction in treatment frequency after receiving 4D-150. This success raises hopes that the gene therapy will similarly benefit DME patients.
Millions of people worldwide suffer from DME or wet AMD. If approved, 4D-150 could significantly reduce their treatment burden while preserving their vision.
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