Anthony DeFino , 2025-05-02 17:53:00
Key takeaways:
- The designation was supported by to-date results of the ongoing SPECTRA study in diabetic macular edema.
- The gene therapy is designed to offer sustained delivery of anti-VEGF after one injection.
The FDA granted regenerative medicine advanced therapy designation to 4D-150 for the treatment of diabetic macular edema, according to a press release from 4D Molecular Therapeutics.
The gene therapy is designed to offer sustained delivery of anti-VEGF (aflibercept and anti-VEGF-C) with a single intravitreal injection. The regenerative medicine advanced therapy (RMAT) designation is granted with the intent of expediting the development and review of therapies, offering sponsor companies the benefits of fast track and breakthrough therapy designations, the release said.
The designation was supported by to-date results of the ongoing SPECTRA study in DME, according to David Kirn, MD, co-founder and CEO of 4DMT.
“This designation in DME follows the RMAT designation granted for 4D-150 in wet AMD, and to our knowledge, 4D-150 is the first investigational medicine to be granted the designation in both indications,” he said. “We look forward to continuing our ongoing collaboration with the FDA to advance 4D-150 into phase 3 development with an aligned-upon single phase 3 trial for approval in DME, combined with our two wet AMD 4FRONT phase 3 clinical trials.”
Perspective
We learned on May 1 that 4DMT was granted regenerative medicine advanced therapy designation by the FDA for the continued investigation and development of 4D-150 as a treatment of diabetic macular edema using its novel genetic medicine approach to this ubiquitous and vision-threatening disease. This was on the basis of the review of its results to date from the 4D-150 SPECTRA DME clinical trial and follows its prior receipt of the same designation for its wet age-related macular degeneration regulatory therapeutic development program on the basis of the two wet AMD 4FRONT phase 3 pivotal studies.
These are important achievements insofar as this designation has the potential to allow for an expedited drug development pathway for both diseases — a unique accomplishment in our field. Like wet AMD, effective DME management requires not only potent and effective intraocular anti-VEGF pharmacotherapy but all too frequent exposure to the drug — typically requiring six or more injections per year using standard treatment approaches. The 4DMT candidate agent for DME (4D-150) has the potential to have a just a single injection accomplish the same goal. This promising therapy was developed based upon the company’s proprietary Therapeutic Vector Evolution platform and evolved intravitreal vector, R100.
Andrew A. Moshfeghi, MD, MBA
Healio | OSN Retina/Vitreous Section Editor
Associate professor of clinical ophthalmology
Vice chair for clinical affairs
Medical director, USC Roski Eye Institute
Disclosures: Moshfeghi reports no relevant financial disclosures.
